recommendations on new drugs from the lothian … · *for smc advice relating to the use of...

09 September 2020

Produced by the Medicines Management Team, NHS Lothian - email [email protected]

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Recommendations from the Lothian Formulary Committee (FC) following Scottish Medicines Consortium (SMC) advice, NICE MTA advice,

(FAF3) unlicensed and off-label medicines and (FAF2) medicines not considered by SMC

8 - Malignant Disease and Immunosuppression

In alphabetical order Product

Manufacturer

Date SMC/NICE Recommendation Report number

Condition being treated

For more details see www.scottishmedicines.org.uk

NHS Lothian decision

Date of NHS Lothian

decision

5-aminolevulinic acid hydrochloride

(Gliolan®)

Medac

Local formulary process

To help guide resection of glioblastomas.

Added to the Additional List, for Specialist

Use only.

December 2013

5-aminolaevulinic acid (as hydrochlorid e )

78mg/g gel (Ameluz®)

Biofrontera Bioscience GmbH

12.02.18

SMC Report No. 1260/17 RESUBMISSION

5-aminolaevulinic acid (as hydrochloride) (Ameluz®) is accepted for use within NHS Scotland fo r

treatment of superficial and / or nodular basal cell carcinoma (BCC) unsuitable for surgical treatment due to possible treatment-related morbidity and / or poor cosmetic outcome in adults.

In a phase III study of patients with BCC, up to two cycles of photodynamic therapy (PDT) with 5 aminolaevulinic acid gel was non-inferior to PDT with an alternative photosensitising a g e nt fo r

the primary endpoint, complete clearance, defined as clearance of all treated lesions, assessed visually at 12 weeks after the last PDT.

Not routinely available as local experts d o

not wish to add the medicine to the formulary at this time.

April 2018

mailto:[email protected]

http://www.scottishmedicines.org.uk/

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Product

Manufacturer





Date of NHS Lothian

decision

abemaciclib 50mg, 100mg and 150mg

tablets (Verzenios®)

Eli Lilly and Company

13.05.19

SMC Report No. 2135

abemaciclib (Verzenios®) is accepted for use within NHSScotland for the treatment of women

with hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative locally advanced or metastatic breast cancer in combination with an aromatase

inhibitor* as initial endocrine-based therapy, or in women who have received prior endocrine therapy.

In a phase III randomised study in women with HR-positive, HER2-negative ad va n ced b re ast cancer, abemaciclib in combination with an aromatase inhibitor significantly increased

progression-free survival compared with aromatase inhibitor monotherapy. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of abemaciclib. This advice is contingent upon the continuing availa b il it y of the PAS in NHSScotland or a list price that is equivalent or lower.

*For SMC advice relating to the use of abemaciclib in combination with fulvestrant in this setting, please refer to SMC Report No. 2179

Routinely available in line with national

guidance. Included on the Additiona l L ist for Specialist Use only.

March 2020

abemaciclib 50mg, 100mg and 150mg tablets (Verzenios

®)

Eli Lilly and Company

13.05.19 SMC Report No. 2179

abemaciclib (Verzenios®) is accepted for restricted use within NHSScotland.

Indication under review: For the treatment of women with hormone receptor (HR) positive,

human epidermal growth factor receptor 2 (HER2) negative locally advanced or metastatic breast cancer in combination with fulvestrant* as initial endocrine-based therap y o r in wo me n

who have received prior endocrine therapy. SMC restriction: for use in women who have progressed on or after (neo) adjuva n t e nd o crin e

therapy, or progressed during first-line endocrine-based therapy for advanced breast cancer In a phase III randomised study in women with HR-positive, HER2-negative ad va n ced b re ast

cancer who had received prior endocrine therapy, abemaciclib in combination with fu lve st ra n t significantly increased progression-free survival compared with endocrine monotherapy.

This SMC advice takes account of the benefit of Patient Access Schemes (PAS) th a t imp ro ve the cost effectiveness of abemaciclib and fulvestrant. This advice is contingent upon the

continuing availability of these PAS in NHSScotland or list prices that are equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting. * For SMC advice relating to the use of abemaciclib in combination with an aromatase inh ib ito r

in this setting, please refer to SMC report No. 2135

Routinely available in line with national guidance. Included on the Additiona l L ist

for Specialist Use only.

March 2020



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Product

Manufacturer





Date of NHS Lothian

decision

abiraterone acetate 250mg tablets

(Zytiga®)

Janssen-Cilag Ltd

13.08.12


Patient Access Scheme

abiraterone acetate (Zytiga®) is accepted for restricted use within NHS Scotland with prednisone

or prednisolone for the treatment of metastatic castration resistant prostate cancer (mCRPC) in adult men whose disease has progressed on or after a docetaxel-based chemotherapy regimen.

SMC restriction: abiraterone is accepted for use in patients who have received only one prior chemotherapy regimen.

Abiraterone plus prednisone was associated with significantly improved overall survival compared with placebo plus prednisone in patients with mCRPC previously treated with

docetaxel. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of abiraterone. This SMC advice is contingent upon the con tinuing availability of the patient access scheme in NHS Scotland.

Included on the Additional List, Specia list

Use only for the indication in question.

October 2012


(Zytiga®)

Janssen-Cilag Ltd

12.10.15

SMC Report No. 873/13 INDEPENDENT REVIEW PANEL


abirateroneacetate (Zytiga®) is accepted for use within NHS Scotland.

Indication under review: abiraterone acetate is indicated with prednisone or prednisolone for the treatment of metastatic castration resistant prostate cancer (mCRPC) in adult men who are

asymptomatic or mildly symptomatic after failure of androgen deprivation therapy in whom chemotherapy is not yet clinically indicated.

In a randomised, double-blind phase III study of adult men with chemotherapy-n a ive mCRPC, treatment with abiraterone acetate in combination with corticosteroid was associated with a

statistically significant extended progression-free survival and overall survival when co mp a re d with placebo plus corticosteroid.

This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e cost-effectiveness of abiraterone acetate. This advice is contingent upon the continuing

availability of the patient access scheme in NHS Scotland or a list price that is equivalent or lower.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.

Includedon the Additional List, for

Specialist Use only, for the indication in question.

December 2015


(Zytiga®)

Janssen-Cilag Ltd

13.01.20

SMC Report No. 2215 Patient Access Scheme

abiraterone acetate (Zytiga®) is accepted for use within NHSScotland.

Indication under review: abiraterone acetate with prednisone or prednisolone for the treatment of newly diagnosed high risk metastatic hormone sensitive prostate cancer in adult men in

combination with androgen deprivation therapy. Abiraterone acetate in combination with prednisone and androgen deprivation therapy

demonstrated superiority over androgen deprivation therapy alone for improving p ro g re ssio n -free survival and overall survival.

This advice applies only in the context of an approved NHSScotland Patient Acce ss Sch eme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based,

or a PAS/ list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.



March 2020

afatinib 20mg, 30mg, 40mg, 50mg film-coated tablets (Giotrif

®)

Boehringer Ingelheim International GmbH

10.03.14 SMC Report No 920/13


afatinib (Giotrif®) is accepted for use within NHS Scotland as monotherapy, for the treatme nt o f

epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor-naïve adult patients with

locally advanced or metastatic non-small cell lung cancer (NSCLC) with activating EGFR mutation(s).

In two phase III studies, in patients with EGFR mutation positive adenocarcinoma o f th e lu n g , afatinib was significantly superior to the chemotherapy regimen comparators for the primary

endpoint of progression free survival. Overall survival data are immature. A mixed treatment comparison provides indirect comparative data versus other tyrosine kinase inhibitors.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of afatinib. This SMC advice is contingent upon the continuing availabilit y

of the Patient Access Scheme in NHS Scotland or a list price that is equivalent or lower.

For patients with a specific EGFR mutation, exon 19 deletion:

Included on the Additional List, Specia list Use only for the indication in question.

For all other indications:

Not included on the LJF because the NHS Lothian decision is that the medicine does

not represent sufficient added benefit to other comparator medicines to treat the

condition in question.

October 2014

February 2014



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Product

Manufacturer





Date of NHS Lothian

decision

afatinib (Giotrif®) 20 mg/30 mg/40 mg/50

mg film-coated tablets Boehringer Ingelheim Limited

11.07.16

SMC Report No 1174/16 NON SUBMISSION

NOT RECOMMENDED: afatinib (Giotrif®) is not recommended for use within NHS Scot la n d a s

monotherapy for the treatment of locally advanced or metastatic non small cell lu n g ca n ce r o f squamous histology progressing on or after platinum-based chemotherapy.

The holder of the marketing authorisation has not made a submission to SMC regarding this product in this setting. As a result we cannot recommend its use within NHSScotland.

NOT RECOMMENDED

aflibercept 25mg/mL concentrate for

solution for infusion (Zaltrap®)

Sanofi

10.03.14

SMC Report No 878/13 RESUBMISSION


aflibercept (Zaltrap®) is accepted for use within NHS Scotland in combination with irinote ca n /5-

fluorouracil/folinic acid (FOLFIRI) chemotherapy, aflibercept is indicated in adults with metastatic colorectal cancer (mCRC) that is resistant to or has progressed after an oxaliplatin-co n tain in g

regimen. In one randomised, double-blind, phase III study, aflibercept plus FOLFIRI chemotherapy

regimen resulted in statistically significant longer overall survival compared with placebo plus FOLFIRI chemotherapy regimen. However, the effect was of relatively modest clinical benefit.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of aflibercept. This SMC advice is contingent upon the continuing




April 2014

alectinib hydrochloride (Alecensa®)

150mg hard capsules Roche Products Ltd

08.05.17

SMC Report No. 1257/17 NON SUBMISSION

NOT RECOMMENDED: alectinib hydrochloride (Alecensa®) is not recommended for use with in

NHS Scotland. Indication under review: As monotherapy for the treatment of adult patients with anaplastic

lymphoma kinase positive advanced non-small cell lung cancer previously treated with crizotinib. The holder of the marketing authorisation has not made a submission to SMC regarding this

product in this indication. As a result we cannot recommend its use within NHSScotland.

NOT RECOMMENDED

alectinib hydrochloride (Alecensa®)

150mg hard capsules

Roche Products Ltd

13.08.18 SMC Report No.2012


Following a full submission assessed under the orphan medicine process, alectinib (Alecensa®)

is accepted for use within NHS Scotland as monotherapy for the first-line treatment of adult

patients with anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lu n g ca n ce r (NSCLC).

Alectinib, compared with another tyrosine kinase inhibitor, significantly improved progression -free survival in treatment-naïve adults with advanced or recurrent ALK-positive NSCLC.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of alectinib. This advice is contingent upon the continuing availa b il it y o f

the PAS in NHS Scotland or a list price that is equivalent or lower.

Routinely available in line with national guidance. Included on the Additional List,


December 2018

alemtuzumab, 30mg/mL for concentrate for solution for infusion (MabCampath

®)

Bayer plc, Bayer Schering Pharma Division

08.09.08

SMC Report No. 494/08

alemtuzumab (MabCampath®) is accepted for restricted use within NHS Scotland for trea tme n t

of patients with B-cell chronic lymphocytic leukaemia (B-CLL) for whom fludarabine combination

chemotherapy is not appropriate It is restricted to use in patients with previously untreated B-CLL, with the cytogenetic

abnormality 17p-deletion. Compared with an alkylating agent, alemtuzumab was associated with improved progression-free survival in patients with B-CLL. Data in

patients with 17p-deletion are limited; improved survival was demonstrated in a sub-group analysis in 21 patients.

Added to the Additional List, for Specialist use only.

Categorised RED under the ADTC ‘Po licy

for the use of unlicensed (and off-label use) Medicines in NHS Lothian’.

May 2011

alemtuzumab (MabCampath®)

Genzyme


Immunosuppression for islet cell transplant recipients


Use only.

Categorised RED under the ADTC ‘Po licy for the use of unlicensed (and off-label

use) Medicines in NHS Lothian’. Specialist Use only.

January 2011



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Product

Manufacturer





Date of NHS Lothian

decision

alemtuzumab (MabCampath®)

Genzyme


Treatment of relapsed chronic lymphocytic leukaemia (subcutaneous administration)


Use only.



April 2012

anakinra (Kineret®) 100mg solution for

injection in a pre-filled syringe Swedish Orphan Biovitrum Ltd

07.12.15

SMC Report No.1116/15 NON SUBMISSION

NOT RECOMMENDED: anakinra (Kineret®) is not recommended for use within NHS Scotland.

Indication under review:Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS) in adults, adolescents, children and infants aged 8 months and older with a body weight of 10 kg or

above, including:

• Neonatal-Onset Multisystem Inflammatory Disease (NOMID) / Chronic Infantile

Neurological, Cutaneous, Articular Syndrome (CINCA)

• Muckle-Wells Syndrome (MWS) Familial Cold Autoinflammatory Syndrome (FCAS)

The holder of the marketing authorisation has not made a submission to SMC regarding this product in this indication. As a result we cannot recommend its use within NHSScotland.

NOT RECOMMENDED

anastrozole 1mg tablets (Arimidex®)

AstraZeneca UK Ltd

12.09.05 SMC Report No. 198/05

anastrozole (Arimidex) is accepted for restricted use within NHS Scotland in the adjuvant

treatment of postmenopausal women with hormone receptor-positive early invasive breast cancer.

Anastrozole has shown benefit over standard anti-oestrogen therapy in terms of d ise a se -f re e survival in this patient group. It offers an alternative to tamoxifen and has a diffe re n t a d ve rse

effects profile. Treatment with anastrozole should be initiated by a breast cancer specialist.

Added to LJF as second choice treatmen t

for patients at risk of early recurrence or with contraindication to tamoxifen.


guidance. Included in the LJF, for Specialist Initiation.

August 2010

As per LJF

July 2019

anastrozole 1mg tablet (Arimidex®)

AstraZeneca UK Limited

13.11.06 SMC Report No. 322/06

anastrozole (Arimidex®) is accepted for restricted use within NHS Scotland for the adjuvant

treatment of early breast cancer in hormone receptor positive postmenopausal women who have received 2 to 3 years of adjuvant tamoxifen.

In a combined analysis of two trials, switching to anastrozole after 2 years of tamoxifen the ra py rather than continuing with tamoxifen resulted in a 3.1% increase in event-free survival at th re e

years follow-up. It offers an alternative to tamoxifen after initial adjuvant treatment with tamoxifen for 2-3 years and has a different adverse effects profile. Treatment with anast ro zo le

is restricted to initiation by a breast cancer specialist.

‘Not preferred’ in Lothian as suitable

alternatives exist.

August 2010

apalutamide 60mg film-coated tablets

(Erleada®)

Janssen-Cilag Ltd

10.02.20

SMC Report No. 2268 NON SUBMISSION

NOT RECOMMENDED: apalutamide (Erleada®) is not recommended for use within

NHSScotland. Indication under review: In adult men for the treatment of non-metastatic castration-resistant

prostate cancer (NM-CRPC) who are at high risk of developing metastatic disease. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

aprepitant (Emend®)

Merck, Sharpe & Dohme

08.11.04 SMC Report No. 132/04

aprepitant (Emend®) is accepted for restricted usewithin NHS Scotland for the prevention of

acute and delayed nausea and vomiting associated with highly emetogenic cisplatin -based chemotherapy.

The antiemetic regimen incorporating aprepitant was superior to one regimen (where dexamethasone alone was used in the delayed phase of treatment), for the prevention of

cisplatin-induced nausea and vomiting in the acute and delayed phases. It should be in it ia te d only by appropriate hospital based specialists.


Use only.

July 2008



09 September 2020


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Product

Manufacturer





Date of NHS Lothian

decision

aprepitant 80mg, 125mg hard capsule s

(Emend®)

Merck Sharp and Dohme Ltd

07.11.11


NOT RECOMMENDED: aprepitant (Emend®) as part of combination therapy is not

recommended for use within NHS Scotland for prevention of nausea and vomiting a sso cia ted with moderately emetogenic cancer chemotherapy.

Compared with a control regimen, aprepitant has been shown to increase the proportion of patients achieving a complete response in a study of breast cancer patients or experiencin g n o

vomiting in patients with a range of tumour types, when patients were initiated on their first cycle of a moderately emetogenic chemotherapy regimen. However the control regimen was

considered suboptimal for the treatment of delayed symptoms and evidence for use in subsequent cycles is limited.

Overall the submitting company did not present sufficiently robust clinical and economic analyses to gain acceptance by SMC.

NOT RECOMMENDED

aprepitant, 80mg, 125mg hard capsules

and 125mg powder for oral suspension (Emend

®)

Merck, Sharpe & Dohme

10.07.17 SMC Report No. 1241/17

aprepitant (Emend®) is accepted for use within NHS Scotland.

Indication under review: As part of combination therapy, for the prevention of nausea and vomiting associated with moderately emetogenic cancer chemotherapy in infants, toddle rs a n d

children from the age of six months to less than 12 years (powder for oral suspension ) and adolescents from the age of 12 years to 17 years (hard capsules).

A randomised, double-blind, placebo-controlled study demonstrated that the addition of aprepitant to a 5HT3 receptor antagonist (+/- steroid) in children and adolescents receiving

chemotherapy with a moderate to very high emetogenic risk produced a significant anti -e me t ic benefit.


guidance. Included on the Additional L ist , for Specialist Use only.

July 2017

aprepitant (Emend®) 80mg,125mg hard

capsules aprepitant (Emend

®) 125mg powder for

oral suspension Merck Sharp & Dohme Limited

10.07.17

SMC Report No. 1252/17 PRODUCT UPDATE

ABBREVIATED SUBMISSION

aprepitant (Emend®) is accepted for use within NHS Scotland.

Indication under review: As part of combination therapy, for the prevention of nausea and vomiting associated with highly emetogenic cancer chemotherapy in children, toddlers and

infants from the age of six months to <12 years (powder for oral suspension) and a d o lesce n ts from the age of 12 years to 17 years (hard capsules).

SMC has previously accepted aprepitant for the prevention of acute and delayed n a use a a n d vomiting associated with highly emetogenic cisplatin-based chemotherapy in adults. The

marketing authorisation has since been extended to cover prevention of nausea and vomiting in adults associated with highly emetogenic non-cisplatin based chemotherapy. SMC does not plan

to assess this minor licence extension.



July 2017

arsenic trioxide 1mg/mL concentrate for

solution for infusion (Trisenox®)

Teva UK Ltd

14.01.19

SMC Report No. 2025

NOT RECOMMENDED: following a full submission assessed under the orphan equivalent

process arsenic trioxide (Trisenox®) is not recommended for use within NHSScotland in

combination with all-trans-retinoic acid (ATRA [tretinoin]) for the induction of remission, and

consolidation in adult patients with newly diagnosed, low-to-intermediate risk acute promyelocytic leukaemia (APL) (white blood cell count, ≤10 x 103/μl), characterised by the

presence of the t(15;17) translocation and/or the presence of the Pro -Myelocytic Leukaemia/Retinoic-Acid-Receptor-alpha (PML/RAR-alpha) gene.

NOT RECOMMENDED

arsenic trioxide 1mg/mL concentrate for

solution for infusion (Trisenox®)

Teva UK Ltd

08.07.19

SMC Report No. 2181 RESUBMISSION

arsenic trioxide (Trisenox®)is accepted for use within NHSScotland.

Indication under review: in combination with all-trans-retinoic acid (ATRA [tretinoin]) for the induction of remission, and consolidation in adult patients with newly diagnosed, low-to-

intermediate risk acute promyelocytic leukaemia (APL) (white blood cell count ≤10 x 103/µl),

characterised by the presence of the t(15;17) translocation and/or the presence of the

Pro-Myelocytic Leukaemia/Retinoic-Acid-Receptor-alpha (PML/RAR-alpha) gene. In a Phase III study in patients with newly diagnosed, low-to-intermediate risk APL, arsenic

trioxide was non-inferior to anthracycline-based chemotherapy (both in combination with tretinoin) measured by event-free survival. A significant difference in overall survival fa vo u rin g

arsenic trioxide was also demonstrated.


guidance. Included on the Additional L ist for Specialist Use only.

July 2020



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Product

Manufacturer





Date of NHS Lothian

decision

atezolizumab 1,200mg concentrate for

solution for infusion (Tecentriq®)

Roche Products Ltd

12.03.18


NOT RECOMMENDED: atezolizumab (Tecentriq®) is not recommended for use within NHS

Scotland. Indication under review: As monotherapy for the treatment of adult patients with locally

advanced or metastatic urothelial carcinoma after prior platinum-containing chemotherapy or who are considered cisplatin ineligible.

In a single arm, open-label, phase II study of patients with locally advanced or metastatic urothelial carcinoma who had received no previous treatment for metastatic dise a se a n d wh o

were ineligible for cisplatin therapy, treatment with atezolizumab resulted in an objective response in 19% of patients.

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust clin ica l a n d

economic analysis to gain acceptance by SMC. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.

NOT RECOMMENDED

atezolizumab 1,200mg concentrate for solution for infusion (Tecentriq

®)

Roche Products Limited

08.06.18

SMC Report No. 1336/18 Patient Access Scheme

atezolizumab (Tecentriq®) is accepted for restricted use within NHS Scotland

Indication under review: As monotherapy for the treatment of adult patients with locally

advanced or metastatic nonsmall cell lung cancer (NSCLC) after prior chemotherapy. Pa t ie nts with epidermal growth factor receptor (EGFR) activating mutations or anaplastic lymphoma

kinase (ALK)positive tumour mutations should also have received targeted therapy before receiving atezolizumab.

SMC restriction: treatment with atezolizumab is subject to a two-year clinical stopping rule. Atezolizumab, compared with a standard taxane monotherapy, significantly improved overall

survival in adults with advanced NSCLC who had progressed after platinum-based chemotherapy.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of atezolizumab. This advice is contingent upon the continuing availability

of the PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.

Routinely available in line with national guidance. Added to the Additional List, fo r

Specialist Use only.

April 2019



Roche Products Ltd

12.11.18

SMC Report No. 2103

NOT RECOMMENDED: atezolizumab (Tecentriq®) is not recommended for use within

NHSScotland. Indication under review: As monotherapy for the treatment of adult patients with locally

advanced or metastatic urothelial carcinoma after prior platinum-containing chemotherapy. In a phase III randomised study of patients with locally advanced or metastatic urothelial

carcinoma after prior platinum-containing chemotherapy, there was a small numerical in cre a se in median overall survival for patients treated with atezolizumab compared with chemotherapy.

NOT RECOMMENDED



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Product

Manufacturer





Date of NHS Lothian

decision



Roche Products Ltd

11.11.19

SMC Report No. 2208


NHSScotland.

Indication under review: In combination with bevacizumab, paclitaxel and carboplatin, for the first-line treatment of adult patients with metastatic non-squamous non-small cell lung cancer

(NSCLC). In patients with epidermal growth factor receptor (EGFR) mutant or anaplastic lymphoma kinase (ALK)-positive NSCLC, atezolizumab in combination with bevacizumab,

paclitaxel and carboplatin, is indicated only after failure of appropriate targeted therapies. In a phase III study, the addition of atezolizumab to bevacizumab, carboplatin and paclitaxel was

associated with an increase in median progression-free survival in patients with metastatic no n -squamous NSCLC.

The submitting company did not present a sufficiently robust clinical and economic an a lysis to

gain acceptance by SMC.

This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.

NOT RECOMMENDED

atezolizumab 1,200mg concentrate for solution for infusion (Tecentriq

®)

Roche Products Ltd

09.12.19 SMC Report No. 2254

NON SUBMISSION


NHSScotland.

Indication under review: In combination with nab-paclitaxel and carboplatin for the first-line treatment of adult patients with metastatic non-squamous non-small cell lung cance r (NSCL C)

who do not have EGFR mutant or ALK-positive NSCLC. The holder of the marketing authorisation has not made a submission to SMC regarding this

product in this setting. As a result we cannot recommend its use within NHSScotland.

NOT RECOMMENDED

avelumab 20mg/mL concentrate for

solution for infusion (Bavencio®)

Merck Serono Europe Limited/Pfizer

07.05.18


Following a full submission considered under the ultra-orphan and end of life process, avelumab

(Bavencio®) is accepted for use within NHS Scotland.

Indication under review: As monotherapy for the treatment of adult patients with metastatic

Merkel cell carcinoma (mMCC). An uncontrolled phase II study demonstrated that treatment with avelumab for patients with

mMCC who had received prior chemotherapy produced improvements in objective response rate, duration of response and overall survival compared with historical chemotherapy co nt ro ls

from a retrospective cohort study This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.


guidance. Included on the Additional List , for Specialist Use only.

July 2018

axitinib (Inlyta®)

Pfizer


As a second-line treatment for advanced/metastatic renal carcinoma after pazaponib therapy. Added to the Additional List, for Specialist

Use only.



April 2014

axitinib, 1mg and 5mg, film-coated tablets

(Inlyta®)

Pfizer

11.11.13



axitinib (Inlyta®) is accepted for use within NHS Scotland for the treatment of adult patients with

advanced renal cell carcinoma (RCC) after failure of prior treatment with sunitinib or a cytokine. In a phase III, open-label study, axitinib improved progression-free survival significantly more

than another targeted therapy when used after first-line sunitinib or a cytokine. Th e re wa s n o significant improvement in overall survival.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of axitinib. This SMC advice is contingent upon the continuing availabil it y

of the patient access scheme in NHS Scotland or a list price that is equivalent or lower.



Additional tablet strengths are now

available. 1mg, 3mg, 5mg and 7mg tablets are all

included in the Patient Access Scheme.

December 2013

August 2015



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Product

Manufacturer





Date of NHS Lothian

decision

axicabtagene ciloleucel 0.4 – 2 x 108 cells

dispersion for infusion dispersion for infusion (Yescarta

®)

Kite Pharma, a Gilead Company

07.10.19 SMC Report No. 2189

RESUBMISSION Patient Access Scheme

axicabtagene ciloleucel (Yescarta®) is accepted for use within NHSScotland.

Indication under review: Treatment of adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) and primary mediastinal large B cell lymphoma (PMBCL), after two or

more lines of systemic therapy. Axicabtagene ciloleucel was associated with an objective response rate of 82% in a single-arm,

open-label, phase I/II study in patients with refractory DLBCL. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of axicabtagene ciloleucel. This advice is contingent upon the continuing availability of the PAS in NHSScotland or a list price that is equivalent or lower.



guidance. Included on the Additional L ist for Specialist Use only..

December 2019

azacitidine 100mg powder for suspension

for injection (Vidaza®)

Celgene Ltd

12.09.11



azacitidine (Vidaza®) is accepted for use within NHS Scotland for treatment of adult patients who

are not eligible for haematopoietic stem cell transplantation (SCT) with intermediate -2 and high -risk myelodysplastic syndrome (MDS), chronic myelomonocytic leukaemia (CMML) or acute

myeloid leukaemia (AML). Azacitidine therapy produced a significant increase in overall survival compared with

conventional care regimens in previously untreated higher-risk MDS patients. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of azacitidine. This SMC advice is contingent upon the continuing availability of the PAS in NHS Scotland.


Use only.

November 2011

azacitidine (Vidaza®) 25 mg/mL powder

for suspension for injection Celgene Ltd

11.07.16

SMC Report No: 1175/16 NON SUBMISSION

NOT RECOMMENDED: azacitidine (Vidaza®) is not recommended for use within NHS Scotlan d

for the treatment of adult patients aged 65 years or older who are not eligible for haematopoietic stem cell transplantation (HSCT) with acute myeloid leukaemia (AML) with >30% marrow bla sts

according to the World Health Organisation (WHO) classification. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

basiliximab (Simulect®)

Novartis


For prevention of acute organ rejection in liver transplant in patients with or at risk of renal dysfunction to allow delayed introduction of calcineurin inhibitors,

in order to reduce incidence of acute renal inpairment.

Not recommended for use in Lothian. Categorised BLACKunder the ADTC

‘Policy and procedures for the use of unlicensed medicines’, for the prevention

of acute organ rejection in liver transplant.

November 2014

belatacept powder for concentrate for solution for infusion 250mg vial and

disposable syringe (Nulojix®)

Bristol Myers Squibb Pharmaceuticals Ltd

11.06.12


NOT RECOMMENDED: belatacept (Nulojix®) is not recommended for use within NHS Scotla n d

in combination with corticosteroids and mycophenolic acid, is indicated for prophylaxis o f g ra f t

rejection in adults receiving a renal transplant. It is recommended to add an interleukin -2 receptor antagonist for induction therapy to this belatacept-based regimen.

Results of two phase III studies have demonstrated comparable graft and patient survival of belatacept versus a calcineurin inhibitor when used as part of a maintenance

immunosuppressive regimen. Indirect efficacy data from a mixed treatment comparison are available for belatacept versus another calcineurin inhibitor, considered the key comp a ra to r in

NHS Scotland. The submitting company’s justification for the treatment’s cost in relation to its hea l t h b e ne f it s

was not sufficient and in addition, the company did not present a sufficiently ro b u st e co no mic case to gain acceptance by SMC.

NOT RECOMMENDED



09 September 2020


10/97

Product

Manufacturer





Date of NHS Lothian

decision

belimumab, 120mg and 400mg powder

for concentrate for solution for infusion (Benlysta

®)

GlaxoSmithKline

08.05.17 SMC Report No.775/12


belimumab (Benlysta®) is accepted for restricted use within NHS Scotland.

Indication under review: Add-on therapy in adult patients with active, autoantibody-positive systemic lupus erythematosus (SLE) with a high degree of disease activity (e.g. positive anti-

dsDNA and low complement) despite standard therapy. SMC restriction: patients with evidence of serological disease activity (i.e. positive a n t i-d sDNA

and low complement) and a Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus Disease Activity Index (SELENA-SLEDAI) score ≥10.

Belimumab, in addition to standard of care, modestly improved disease control in patie nts with SLE in two phase III studies.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of belimumab. This advice is contingent upon the continuing availability of




August 2017

bendamustine hydrochloride 25mg, 100mg powder for solution for infusion

(Levact®)

Napp Pharmaceuticals Limited

11.04.11


bendamustine hydrochloride (Levact®) is accepted for use within NHS Scotland.

Indication under review: first-line treatment of chronic lymphocytic leukaemia (CLL) (Binet stag e

B or C) in patients for whom fludarabine combination chemotherapy is not appropriate. Bendamustine showed significantly improved response rates and progression free survival when

compared with another alkylating agent in patients with previously untreated advanced CLL, although the patients studied may have been younger and fitter than those eligib le t o re ce ive

bendamustine in Scottish clinical practice.

Added to the Additional List.

Included on the Lothian Joint Formulary for the indication in question.

Until further evidence is provided, the u se

of bendamustine in combination with rituximab can be undertaken via non-

formulary route.

April 2012

bendamustine 2.5mg/mL powder for concentrate for solution for infusion

(Levact®)


11.04.11


NOT RECOMMENDED: bendamustine (Levact®) is not recommended for use within NHS

Scotland.

Indication under review: for the front line treatment of multiple myeloma (Durie -Salmon sta g e I I with progress or stage III) in combination with prednisone for patients older than 65 ye a rs wh o

are not eligible for autologous stem cell transplantation and who have clinical neuropathy at time of diagnosis precluding the use of thalidomide or bortezomib containing treatment.


NOT RECOMMENDED

bendamustine 2.5mg/mL powder for concentrate for solution for infusion

(Levact®)


11.04.11


NOT RECOMMENDED: bendamustine (Levact®) is not recommended for use within NHS

Scotland.

Indication under review: for the front line treatment of indolent non-Hodgkin's lymphomas as monotherapy in patients who have progressed during or within 6 months following treatment with

rituximab or a rituximab containing regimen. The holder of the marketing authorisation has not made a submission to SMC regarding this

product in this indication. As a result we cannot recommend its use within NHS Scotland.

NOT RECOMMENDED

bendamustine (Levact®)

NAPP


In combination with rituximab for relapsed chronic lymphocytic leukaemia.


Use only.


use) Medicines in NHS Lothian’.

February 2014



09 September 2020


11/97

Product

Manufacturer





Date of NHS Lothian

decision

bendamustine 2.5mg/mL powder for

concentrate for solution of infusion vials (Levact

®) (available as 25mg and 100 mg

vials) Napp Pharmaceuticals


Relapsed and refractory multiple myeloma. Routinely available in line with local or

regional guidance. Included on the Additional List, for

Specialist Use only. Categorised RED under the ADTC ‘Po licy


April 2017

bevacizumab 100mg/4mL and

400mg/16mL solution for intravenous infusion (Avastin

®)

Roche

January 2012 NICE MTA 242 supersedes SMC Report

No. 221/05

NOT RECOMMENDED: NICE MTA 242Bevacizumab in combination with non-oxaliplatin

(fluoropyrimide-based) chemotherapy is not recommended for the treatment of people with metastatic colorectal cancer that has progressed after first-line chemotherapy.

NOT RECOMMENDED

bevacizumab (Avastin®)

Roche Pharmaceuticals

09.07.07


NOT RECOMMENDED: bevacizumab (Avastin®) in combination with paclitaxel is not

recommended for first-line treatment of patients with metastatic breast cancer.


NOT RECOMMENDED



10.12.07


NOT RECOMMENDED: bevacizumab (Avastin®) in addition to platinum-based chemotherapy, is

not recommended for first-line treatment of patients with unresectable advanced, metasta tic o r

recurrent non-small cell lung cancer other than predominantly squamous cell histology. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED



10.03.08 SMC Report No. 459/08

NON SUBMISSION

NOT RECOMMENDED: bevacizumab (Avastin®) is not recommended for use within NHS

Scotland in combination with interferon alfa-2a for the first line treatment of patients with advanced and/or metastatic renal cell cancer.

The holder of the marketing authorisation has not made a submission to SMC regarding this product in this indication. As a result we cannot recommend its use within NHS Scotland.

NOT RECOMMENDED

bevacizumab, 100mg and 400mg vials

(Avastin)

Roche

09.06.08 SMC Report No.469/08

NOT RECOMMENDED: bevacizumab (Avastin) is not recommended for use within NHS Scotland in combination with fluoropyrimidine-based chemotherapy for treatment of patients with

metastatic carcinoma of the colon or rectum. In a randomised trial standard chemotherapy plus bevacizumab showed a small benefit over

standard chemotherapy alone in terms of progression-free survival. However, the manufacturer did not present a sufficiently robust economic analysis to gain acceptance by SMC.

NOT RECOMMENDED



09 September 2020


12/97

Product

Manufacturer





Date of NHS Lothian

decision

bevacizumab, 25mg/mL, concentrate fo r

solution for infusion (Avastin®)

Roche Products Ltd.

14.05.12

SMC Report No.778/12


Scotland in combination with capecitabine is indicated for first-line treatment of patients with metastatic breast cancer in whom treatment with other chemotherapy options including taxan es

or anthracyclines is not considered appropriate. In a double-blind, multicentre, randomised, placebo-controlled phase III study in p a t ie n ts with

locally recurrent or metastatic breast cancer, treatment with bevacizumab plus capecitabine was associated with an extended median progression-free survival of 2.9 months compared with

capecitabine monotherapy. However, there was no overall significant improvement in survival. The submitting company did not present a sufficiently robust economic analysis and, in addition,

their justification of the treatment’s cost in relation to its health benefits was not sufficient to gain acceptance by the SMC.

NOT RECOMMENDED

bevacizumab, 25mg/mL, concentrate fo r


Roche Products Ltd

09.11.15

SMC Report No.806/12 2

nd RESUBMISSION

bevacizumab (Avastin®) is accepted for restricted use within NHS Scotland.

Indication under review: In combination with carboplatin and paclitaxel, for the front-line treatment of advanced (International Federation of Gynaecology and

Obstetrics (FIGO) stages IIIB, IIIC and IV) epithelial ovarian, fallopian tube, or primary peritoneal cancer.

SMC restriction: In patients with FIGO stage IV disease Addition of bevacizumab to standard chemotherapy with carboplatin and paclitaxel increased

progression-free survival. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.

Included on the Additional List, for

Specialist Use only. Categorised REDunder the ADTC ‘Policy

for the use of unlicensed (and off-label use) Medicines in NHS Lothian’, for the

indication in question.

Off label dose being used.

April 2016

bevacizumab, 25mg/mL, concentrate fo r solution for infusion (Avastin

®)

Roche Products Ltd

11.03.13 SMC Report No.

853/13


Scotland in combination with carboplatin and gemcitabine, is indicated for treatment of adult

patients with first recurrence of platinum-sensitive epithelian ovarian, fallopian tub e o r p rima ry peritoneal cancer who have not received prior therapy with bevacizumab or other VEGF

inhibitors or VEGF receptor–targeted agents. A randomised double-blind, placebo-controlled, phase III study demonstrated a significant

improvement in progression-free survival (PFS) in patients with platinum-sensitive recurrent ovarian cancer (ROC) treated with bevacizumab in combination with gemcitabine and

carboplatin, compared with gemcitabine and carboplatin alone. The submitting company's justification of the treatment's cost in relation to its health benefits

was not sufficient to gain acceptance by SMC.

NOT RECOMMENDED

bevacizumab 25mg/mL concentrate for

solution for infusion, (Avastin®)

Roche Products Ltd

07.09.15



Indication under review: in combination with paclitaxel, topotecan, or pegylated liposomal doxorubicin for the treatment of adult patients with platinum-resistant recurrent epithelial ovarian,

fallopian tube, or primary peritoneal cancer who received no more than two prior chemotherapy regimens and who have not received prior therapy with bevacizumab or other vascular

endothelial growth factor (VEGF) inhibitors or VEGF receptor-targeted agents. SMC restriction: to use in combination with paclitaxel.

The addition of bevacizumab to chemotherapy improved progression free survival in patients with platinum-resistant ovarian cancer in an open-label phase III randomised study.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of bevacizumab. This advice is contingent upon the continuing availability

of the PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.



December 2015



09 September 2020


13/97

Product

Manufacturer





Date of NHS Lothian

decision

bevacizumab 25mg/mL concentrate for


Roche Products Ltd

09.05.16



Indication under review: in combination with paclitaxel and cisplatin or, alternatively, p a clitaxe l and topotecan in patients who cannot receive platinum therapy, for the treatment of adult

patients with persistent, recurrent, or metastatic carcinoma of the cervix. Restriction: for use in combination with cisplatin and paclitaxel.

In an open-label, randomised, phase III study, the addition of bevacizumab to combination chemotherapy increased overall survival.

This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e cost effectiveness of bevacizumab. This advice is contingent upon the continuing availabil it y o f

the patientaccess scheme in NHS Scotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician and Engag eme n t (PACE)

meeting.


Use only, for the indication in question.

August 2016

bevacizumab (Avastin®) 25 mg/mL

concentrate for solution for infusion

Roche Products Ltd

12.09.16 SMC Report No. 1190/16

NON SUBMISSION


Scotland in combination with erlotinib for first-line treatment of adult patients with unrese ctab le

advanced, metastatic or recurrent non-squamous non-small cell lung cancer with Epidermal Growth Factor Receptor (EGFR) activating mutations.


NOT RECOMMENDED

bevacizumab 25mg/mL concentrate for solution for infusion (Avastin

®)

Roche Products Ltd

11.09.17 SMC Report No. 1275/17

NON SUBMISSION


Scotland.

Indication under review: In combination with carboplatin and paclitaxel for the treatment of adult patients with first recurrence of platinum-sensitive epithelial ovarian, fallopian tube or primary

peritoneal cancer who have not received prior therapy with bevacizumab or other VEGF inhibitors or VEGF receptor–targeted agents.


NOT RECOMMENDED

bexarotene capsules (Targretin) Elan Pharma

08.11.02 SMC Report No. 14/02

bexarotene capsules (Targretin) is recommended as a second line treatment for patien ts with advanced (stages Ilb or III) cutaneous T-cell lymphoma. Bexarotene treatment should norma lly be initiated and supervised by haematologists, dermatologists or oncologists and used for

patients who have proved refractory both to local skin directed therapy and to at least one systemic treatment.

Added to the Additional List as a second line treatment for patients with cutan e o us

T-cell lymphoma (stages IIb or III).

May 2003

blinatumomab, 38.5 micrograms powder

for concentrate and solution for solution

for infusion (Blincyto)

Amgen Europe B.V.

13.06.16 SMC Report No. 1145/16


blinatumomab (Blincyto®) is accepted for use within NHS Scotland for the treatment of adults

with Philadelphia chromosome negative relapsed or refractory B-precursor acute lymphobla stic leukaemia (ALL).

In a non-comparative phase II study of patients with relapsed or refractory Philadelphia chromosome-negative B-precursor ALL, blinatumomab was associated with clinically relevant

complete remission rates. Controlled data with clinical outcomes are currently lacking. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of blinatumomab. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.



October 2016



09 September 2020


14/97

Product

Manufacturer





Date of NHS Lothian

decision

blinatumomab 38.5 micrograms powder

for concentrate and solution for solution

for infusion (Blincyto) Amgen Europe B.V.

08.04.19

SMC Report No. 2148 PRODUCT UPDATE

ABBREVIATED SUBMISSION Patient Access Scheme

blinatumomab (Blincyto®) is accepted for use within NHSScotland as monotherapy for the

treatment of paediatric patients aged 1 year or older with Philadelphia chromo so me n e ga t ive CD19 positive B-cell precursor acute lymphoblastic leukaemia which is refractory or in re la p se

after receiving at least two prior therapies or in relapse after receiving prior allogeneic hematopoietic stem cell transplantation.

SMC accepted blinatumomab for use in adults following a submission under the end of life a n d ultra-orphan process.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of blinatumomab. This advice is contingent upon the continuing

availability of the PAS in NHS Scotland or a list price that is equivalent or lower.



April 2019

blinatumomab 38.5 micrograms powder for concentrate and

solution for infusion (Blincyto®)

Amgen Ltd

09.03.20 SMC Report No. 2234


Following a full submission considered under the end of life and orphan equivalent process, blinatumomab (Blincyto

®) is accepted for restricted use within NHSScotland.

Indication under review: As monotherapy for the treatment of adults with Philade lphia chromosome negative, CD19 positive, B-precursor acute lymphoblastic leukaemia (ALL) in f irst

or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%.

SMC restriction: to patients who are in first complete remission with minimal residual disease (MRD) greater than or equal to 0.1%.

In a single arm phase II study of patients with B-cell precursor ALL in first or later complete remission and with persistent or recurrent MRD, blinatumomab was associated with clinically

relevant complete MRD response rates. This advice applies only in the context of an approved NHSScotland Patient Acce ss Sch eme

(PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.


Not routinely available as local clinical experts do not wish to add the medicine to

the formulary at this time or there is a local preference for alternative medicines.

May 2020

bortezomib (Velcade®)

Ortho Biotech

11.10.04


bortezomib (Velcade) is accepted for use within NHS Scotland for the treatment of patients with multiple myeloma who have received at least two prior therapies, have demonstra te d d ise a se progression on the last therapy and who are refractory to alternative licensed treatments for this

stage of the disease. Bortezomib produced a disease response in approximately one third of these patients in a n

open-label uncontrolled study. Any other use of bortezomib should only take place with in th e context of a controlled study.

The manufacturers are encouraged to mount an observational study in collaboration with haemato-oncologists to gain more information on the benefits and risks of this therapy.

Added to the Additional List, for Specialist Use only.

Bortezomib may be appropriate for

patients who have relapsed after thalidomide treatment according to agreed

protocol.

November 2004

bortezomib 3.5mg powder for intravenous injection (Velcade

®)

Ortho Biotech

09.11.09 SMC Report No. 302/06

2ND


bortezomib (Velcade®) is accepted for use within NHS Scotland as mono-therapy for the

treatment of progressive multiple myeloma in patients who have received at least one prior

therapy and who have already undergone or are unsuitable for bone marrow transplantation. Bortezomib, compared to high dose dexamethasone, prolonged time to disease progression and

improved survival in patients who had progressive multiple myeloma despite previous treatmen t with one to three lines of therapy.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of bortezomib. This SMC advice is contingent upon the continuing

availability of the patient access scheme in NHS Scotland.


December 2011



09 September 2020


15/97

Product

Manufacturer





Date of NHS Lothian

decision

bortezomib (Velcade®) 3.5mg powder fo r

subcutaneous injection Janssen-Cilag Ltd

10.12.12



bortezomib subcutaneous injection (Velcade®) is accepted for use within NHS Scotland in

combination with melphalan and prednisone for the treatment of patients with previously untreated multiple myeloma who are not eligible for high-dose chemotherapy with bone marro w

transplant. As monotherapy for the treatment of progressive multiple myeloma in patients who have

received at least one prior therapy and who have already undergone or are unsuitable for b o n e marrow transplantation.

The subcutaneous formulation of bortezomib has been shown to be clinically non-inferior to th e intravenous formulation and is the same price.

SMC previously accepted bortezomib intravenous injection as monotherapy in the treatme n t o f multiple myeloma when the benefits of a Patient Access Scheme (PAS) were taken into

account. The Patient Access Scheme Assessment Group (PASAG) has confirmed that this response-based PAS also applies to the subcutaneous formulation when used in th is se t t in g .

This SMC advice is therefore contingent upon the continuing availability of the pa t ie nt a cce ss scheme in NHS Scotland or a list price that is equivalent or lower.

Bortezomib intravenous injection has also been accepted for use in NHS Scotla n d in sp e cif ic circumstances in the first line treatment of multiple myeloma as Healthcare Improvement

Scotland has endorsed NICE MTA No 228 (Bortezomib and thalidomide for the first line treatment of multiple myeloma) in July 2011. The PAS does not apply to the use of bortezo mib

in this setting.


Specialist Use only, for the indication included in PAS.

December 2012

bortezomib 3.5mg powder for solution fo r injection (Velcade

®)

Janssen-Cilag Ltd

13.01.14 SMC Report No. 927/13

bortezomib (Velcade®) is accepted for restricted use within NHS Scotland in combination with

dexamethasone, or with dexamethasone and thalidomide, for the induction treatme nt o f a d u lt

patients with previously untreated multiple myeloma who are eligible for high-dose chemotherapy with haematopoietic stem cell transplantation.

SMC restriction: use as triple therapy in combination with dexamethasone and thalidomide. Bortezomib, used in combination with dexamethasone and thalidomide for the induction

treatment of adult patients with previously untreated multiple myeloma who are eligible for hig h -dose chemotherapy with haematopoietic stem cell transplantation improved response rates

compared with a dual combination regimen.

Included on the Additional List, for Specialist Use only, for the indication in

question.

February 2014



09 September 2020


16/97

Product

Manufacturer





Date of NHS Lothian

decision

bortezomib 3.5mg powder for solution fo r

injection (Velcade®)

Janssen-Cilag Ltd

07.09.15


bortezomib (Velcade®) is accepted for use within NHS Scotland.

Indication under review: in combination with rituximab, cyclophosphamide, doxorubicin and prednisone for the treatment of adult patients with previously untreated mantle cell lymphoma

who are unsuitable for haematopoietic stem cell transplantation. Bortezomib in combination with rituximab, cyclophosphamide, doxorubicin and prednisone

significantly improved progression-free survival compared to a regimen containing rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone in adults with previously untreated

mantle cell lymphoma who were unsuitable for haematopoietic stem cell transplantation



October 2015

bosutinib 100mg, 500mg film-coated tablets (Bosulif

®)

Pfizer Ltd

09.02.15 SMC Report No. 910/13


bosutinib (Bosulif®) is accepted for use within NHS Scotland for the treatment of adult p a t ie nts

with chronic phase (CP), accelerated phase (AP), and blast phase (BP) Philadelphia

chromosome positive chronic myelogenous leukaemia (Ph+ CML) previously treated with one or more tyrosine kinase inhibitor(s) and for whom imatinib, nilotinib and dasatinib are not

considered appropriate treatment options. Major cytogenetic response was achieved in 23/52 patients who represented “u n me t me d ica l

need” within a non-comparative phase I/II study, in which the full population included 546 patients with CP, AP or BP imatinib pre-treated Ph+ CML.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of bosutinib. This advice is contingent upon the continuing availabil it y o f

the patient access scheme in NHS Scotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.


question.

April 2015

bosutinib 100mg, 500mg film-coated tablets (Bosulif

®)

Pfizer Ltd

13.08.18 SMC Report No. 2109

NON SUBMISSION

NOT RECOMMENDED: bosutinib (Bosulif®) is not recommended for use within NHS Scotland.

Indication under review: Treatment of adult patients with newly diagnosed chronic phase

Philadelphia chromosome-positive chronic myelogenous leukaemia. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED



09 September 2020


17/97

Product

Manufacturer





Date of NHS Lothian

decision

brentuximab vedotin (Adcetris®) 50mg

powder for concentrate for solution for infusion

Takeda UK Ltd

13.10.14 SMC Report No. 845/12

brentuximab vedotin (Adcetris®) is accepted for restricted use within NHS Scotland for the

treatment of adult patients with relapsed or refractory CD30+ Hodgkin lymphoma (HL): 1. following autologous stem cell transplant (ASCT) or

2. following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option and

treatment of adult patients with relapsed or refractory systemic anaplastic large cell lymp h o ma (sALCL).

SMC restriction: treatment of adult patients with relapsed or refractory CD30+ Hodgkin lymphoma (HL):

1. following autologous stem cell transplant (ASCT) or 2. following at least two prior therapies when ASCT or multi-agent chemotherapy is not a

treatment option In an open-label, single-arm study, patients with relapsed or refractory Hodgkin lymphoma

treated with brentuximab vedotin achieved an objective response rate of 75%. Controlle d d a ta with clinical outcomes are currently lacking.


Brentuximab is also indicated for the treatment of adult patients with re lapsed or refractory systemic anaplastic large cell lymphoma (sALCL). SMC cannot recommend use in sALCL as the

company didnot include evidence for use in this indication in its submission.



December 2014

brentuximab vedotin (Adcetris®) 50mg

powder for concentrate for solution for

infusion Takeda UK Ltd

07.05.18


NOT RECOMMENDED: brentuximab vedotin (Adcetris®) is not recommended for use within

NHS Scotland.

Indication under review: Treatment of adult patients with CD30+ Hodgkin lymphoma at increased risk of relapse or progression following autologous stem cell transplant.


NOT RECOMMENDED

brentuximab vedotin 50mg powder for

concentrate for solution for infusion (Adcetris

®)

Takeda UK Ltd

10.06.19 SMC Report No. 2202

NON SUBMISSION

NOT RECOMMENDED: brentuximab vedotin (Adcetris®) is not recommended for use within

NHS Scotland. Indication under review: treatment of adult patients with previously untreated CD30 + S ta g e IV

Hodgkin lymphoma in combination with doxorubicin, vinblastine and dacarbazine. The holder of the marketing authorisation has not made a submission to SMC regarding this

product in this setting. As a result we cannot recommend its use within NHSScotland .

NOT RECOMMENDED

brentuximab vedotin 50mg powder for

concentrate for solution for infusion (Adcetris

®)

Takeda UK Ltd

13.01.20 SMC Report No. 2229


brentuximab vedotin (Adcetris®) is accepted for restricted use within NHSScotland.

Indication under review: The treatment of adult patients with CD30+ cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy.

SMC restriction: for the treatment of patients with advanced CTCL, defined as mycosis fungoides stage IIB and above, primary cutaneous anaplastic large cell lymphoma or Sézary

Syndrome. In an open-label, phase III study in patients with previously treated CD30+ CTCL, the obje ct ive

response rate maintained for at least four months was significantly higher in patients who received brentuximab vedotin than physician’s choice of one of two treatments.

This advice applies only in the context of an approved NHSScotland PAS arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/list p rice

that is equivalent or lower.

Not routinely available as local clinical

experts do not wish to add the medicine to the formulary at this time or there is a local

preference for alternative medicines.

March 2020



09 September 2020


18/97

Product

Manufacturer





Date of NHS Lothian

decision

brigatinib 30mg, 90mg and 180mg film-

coated tablets (Alunbrig®)

Takeda UK Ltd

10.06.19 SMC Report No. 2147


brigatinib (Alunbrig®) is accepted for use within NHSScotland.

Indication under review: as monotherapy for the treatment of adult patients with anaplastic lymphoma kinase (ALK) positive advanced non-small cell lung cancer (NSCLC) previously

treated with crizotinib. Brigatinib was associated with an objective response rate of 56% in a single-arm, o p e n -la b e l,

phase II study in patients with ALK-positive NSCLC who had progressed on first-l in e ta rg e te d treatment with crizotinib.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of brigatinib. This advice is contingent upon the continuing availabil it y o f

the PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting .


experts do not wish to add the medicine to the formulary at this time of there is a loca l

preference for alternative medicines

July 2019

busulfan, 6mg/mL, intravenous

(Busilvex®)

Pierre Fabre Ltd

15.01.07


busulfan for intravenous infusion (Busilvex®) is accepted for use within NHS Scotland as part o f

a combination regimen for conditioning treatment prior to conventional haematopoietic progenitor cell transplantation (HPCT) in paediatric and adult patients. The intravenous

preparation offers advantages to patients over the oral formulation in terms of con ve n ie n ce o f administration and predictability of blood levels.

In adults it should be followed by cyclophosphamide (BuCy2) and in children it should be followed by cyclophosphamide (BuCy4) or by melphalan (BuMel).

‘Not preferred’ in Lothian. A submission

has not been made to FC regarding this product for this indication.

June 2008

cabazitaxel, 60mg concentrate and

solvent for solution for infusion(Jevtana®)

Sanofi

12.12.16

SMC Report No. 735/11 2

nd RESUBMISSION


cabazitaxel (Jevtana®) is accepted for restricted use within NHS Scotland in combination with

prednisone or prednisolone is indicated for the treatment of adult patients with hormone refractory metastatic prostate cancer previously treated with a docetaxel-containing regimen.

SMC restriction: for use in patients who have received at least 225mg/m2 (three cycles) of

docetaxel and have an Eastern Cooperative Oncology Group (ECOG) performance status o f 0

or 1. In an open-label, multicentre, randomised-controlled, phase III study in patients with metasta tic

hormone refractory prostate cancer, treatment with cabazitaxel plus prednisone/pre d niso lo ne was associated with an extended median overall survival of 2.4 months compared with an

alternative chemotherapy regimen. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of cabazitaxel. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.



guidance. Included on the Additional L ist , for Specialist Use only, for the indication in

question.

April 2017

cabozantinib 20mg and 80mg hard

capsules (Cometriq®)

Swedish Orphan Biovitrum Ltd.

09.03.15


NOT RECOMMENDED: cabozantinib 20mg and 80mg hard capsules (Cometriq®) is not

recommended for use within NHS Scotland for the treatment of adult patients with progressive, unresectable locally advanced or metastatic medullary thyroid carcinoma.

In one pivotal, phase III study, cabozantinib was associated with a significant advantage in progression-free survival over placebo. However, the difference between cabozantinib and

placebo did not reach statistical significance in the subgroup of patients with Rearranged during Transfection (RET) negative tumours. The summary of product characteristics therefore notes

that for patients in whom RET mutation status is unknown or is negative, a possible lower benefit should be taken into account before individual treatment decision.

The submitting company did not present a sufficiently robust economic analysis and in addition their justification of the treatment’s cost in relation to its benefits was not sufficient to gain

acceptance by SMC. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.

NOT RECOMMENDED



09 September 2020


19/97

Product

Manufacturer





Date of NHS Lothian

decision

cabozantinib 20mg, 40mg and 60mg film-

coated tablets (Cabometyx®)

Ipsen Ltd.

12.06.17


Following a full submission assessed under the end of life process, cabozantinib (Carbometyx®)

is accepted for use within NHS Scotland. Indication under review: for the treatment of advanced renal cell carcinoma (RCC) in adults

following prior vascular endothelial growth factor. Cabozantinib, compared with a mammalian target of rapamycin (mTOR) inhibitor, significantly

increased progression-free survival in patients with advanced or metastatic RCC who had received at least one previous regimen of VEGF receptor tyrosine kinase inhibitor.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of cabozantinib. This advice is contingent upon the continuing availability

of the PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.



August 2017

cabozantinib, 20mg, 40mg, and 60mg

film-coated tablets (Cabometyx®)

Ipsen Ltd UK

11.02.19

SMC Report No. 2136 (previously SMC Report No. 2095 08.10.18)

RESUBMISSION

NOT RECOMMENDED: cabozantinib (Cabometyx®) is not recommended for use within

NHSScotland for advanced renal cell carcinoma (RCC) in treatment-naïve adults with intermediate or poor risk.

In a phase II study, in treatment-naïve adults with advanced RCC with intermediate or poo r risk as defined by the IMDC risk group categories, cabozantinib was superior to another tyrosine

kinase inhibitor for progression free survival. The submitting company did not present a sufficiently robust economic analysis to gain

acceptance by SMC. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.

NOT RECOMMENDED

cabozantinib 20mg, 40mg and 60mg tablets (Cabometyx

®)

Ipsen Ltd

11.02.19 SMC Report No. 2160

NON SUBMISSION

NOT RECOMMENDED: cabozantinib (Cabometyx®) is not recommended for use within

NHSScotland.

Indication under review: As monotherapy for the treatment of hepatocellular carcinoma in adults who have previously been treated with sorafenib.


NOT RECOMMENDED

canakinumab (Ilaris®) 150 mg/mL, powder

for solution for injection Novartis Pharmaceuticals

08.11.10


NOT RECOMMENDED: canakinumab (Ilaris®) is not recommended for use within NHSScotland.

Indication under review: Cryopyrin-Associated Periodic Syndromes (CAPS) in adults, adolescents and children aged 4 years and older with body weight above 15 kg.

The holder of the marketing authorisation has not made a submission to SMC regarding this product in this indication. As a result SMC cannot recommend its use within NHSScotland.

NOT RECOMMENDED

canakinumab (Ilaris®) 150 mg powder fo r

solution for injection Novartis Pharmaceuticals Ltd

10.06.13


NOT RECOMMENDED: canakinumab (Ilaris®) is not recommended for use within NHS Scotland

for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS) in adults, a d o le sce nts and children aged 2 years and older with body weight of 7.5 kg or above including:

• Muckle-Wells Syndrome (MWS)

• Neonatal-Onset Multisystem Inflammatory Disease (NOMID) / Chronic Infantile Neurological, Cutaneous, Articular Syndrome (CINCA)

• Severe forms of Familial Cold Autoinflammatory Syndrome (FCAS) / Familial Cold Urt ica ria (FCU) presenting with signs and symptoms beyond cold-induced urticarial skin rash

The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED



09 September 2020


20/97

Product

Manufacturer





Date of NHS Lothian

decision

capecitabine (Xeloda)


Treatment of early breast cancer following neoadjuvant anthracycline

and/or taxane based chemotherapy with residual diseasein breast or axillary lymph nodes at time of surgery.

Routinely available in line with local or

regional guidance. Included on the Additional List, for Specialist Use only.



November 2018

capecitabine (Xeloda) Roche

07.03.03


capecitabine (Xeloda) is accepted for restricted use within NHS Scotland. Capecitabine is recommended for use in Scotland by oncologists with appropriate expertise in tre a t in g lo ca lly

advanced/metastatic breast cancer. It is an orally active treatment which has improved outcomes both as monotherapy in those

previously treated with an anthracycline and a taxane, and in combination with docetaxel in those previously treated with an anthracycline.

Included in the Formulary.

capecitabine 150 and 500mg tablets

(Xeloda®)

Roche

05.08.05


capecitabine (Xeloda) is accepted for use within NHS Scotland for the adjuvant t re a tme nt o f patients following surgery for Stage III (Dukes’ C stage) colon cancer. Oral capecitabine

appears to be as least as effective as standard IV 5FU/FA chemotherapy with the convenien ce of oral administration. It should only be prescribed by oncologists.

It is more expensive than IV chemotherapy regimens. However, its use may allow ch a n g e s to service delivery that have individual patient or organisational benefits.


Use only.

October 2005

capecitabine, 150mg and 500mg tablets (Xeloda

®) No. (401/07)

Roche

10.09.07 SMC Report No. 401/07

capecitabine (Xeloda®) is accepted for use within NHS Scotland for first line treatment of

patients with advanced gastric cancer in combination with a platinum-based chemotherapy

regimen. Capecitabine was non-inferior to continuously infused intravenous 5-FU in terms of progression-

free survival when each was used in combination with a platinum-based drug in patients with advanced gastric cancer. It also demonstrated non-inferiority in overall survival comp a re d with

continuously infused intravenous 5-FU in patients with advanced gastric cancer when each wa s used in a triple regimen containing a platinum-based drug and an anthracycline drug.

Capecitabine is more expensive than 5-FU, however, the convenience of oral administration may allow changes to service delivery that have individual patient or organisational benefits.


January 2009

capecitabine 150mg and 500mg tablets

(Xeloda®)


13.10.08


capecitabine 150mg and 500mg tablets (Xeloda®) is accepted for use within NHS Sco t la n d fo r

the treatment of metastatic colorectal cancer. The convenience of oral administration may allow changes to service delivery that have

individual patient or organisational benefits, though these may be lessened when it is used in regimens whose other components require intravenous administration.

Added to the Additional List – for Specialist

Use only.

August 2009

capecitabine, 150mg, 500mg, tablets (Xeloda

®)


08.08.11 SMC Report No. 716/11

capecitabine (Xeloda®) is accepted for use within NHS Scotland.

Indication under review: The adjuvant treatment of patients following surgery of stage III (Dukes’

stage C) colon cancer in combination with oxaliplatin. At 55 months, disease free survival was significantly increased for capecitabine plus oxaliplatin -

treated patients compared with a recognised regimen containing a fluoropyramidine in the adjuvant treatment of patients with completely resected stage III (Dukes’ C) colon cancer.


August 2011

capecitabine (Xeloda®)

Roche

Adjuvant use following potentially curative surgery for adenocarcinoma of the pancreas to

replace 5FU/folinic acid given according to the Mayo schedule

Added to the LJF as first choice for use

post-operatively in the treatment of adenocarcinoma of the pancreas, for


January 2007



09 September 2020


21/97

Product

Manufacturer





Date of NHS Lothian

decision

capecitabine (Xeloda) Roche


Colorectal cancer Added to the Additional List for use in

secondary care only. Specialist Use only.

Categorised RED under the ADTC ‘Policy for the use of unlicensed (and off-label


January 2005

capecitabine (Xeloda®)

Roche


Pre-operative chemoradiation in patients with locally advanced rectal cancer requiring down staging prior to definitive resection or in patients with good

performance status and low volume metastatic disease requiring durable palliation for unresectable rectal cancer




September 2006

capecitabine (Xelox®)

Roche


In combination with epirubicin and cisplatin (ECX) for palliative treatment

of advanced oesphagogastric cancer


Use only. Categorised RED under the ADTC ‘Po licy


September 2005

capecitabine/oxaliplatin (Xeloda

®/Eloxatin

®)

Roche

First choice treatment for Dukes B colorectal cancer Added to the Additional List, for Specialist Use only.

July 2007

carmustine 7.7mg implant (Gliadel®)

Link Pharmaceuticals Ltd

12.12.05 SMC Report No. 215/05

carmustine implant (Gliadel®) is accepted for use within NHS Scotland for the treatment of newly

diagnosed high-grade malignant glioma patients as an adjunct to surgery and radiation. In the pivotal study, the use of carmustine implants was associated with a 29% relative decrease

in the risk of death, which equates to an increase in median survival time of 2.3 months.


Use only.

March 2006

catumaxomab (Removab®) 10 and 50

microgram concentrate for solution for infusion

Fresenius Biotech GmbH

09.04.12 SMC Report No. 788/12

NON SUBMISSION

NOT RECOMMENDED: catumaxomab (Removab®) is not recommended for use within NHS

Scotland for intraperitoneal treatment of malignant ascites in patients with EpCAM-positive carcinomas where standard therapy is not available or no longer feasible.


NOT RECOMMENDED

carfilzomib 60mg powder for solution for infusion (Kyprolis

®)

Amgen Ltd.

16.01.17 SMC Report No. 1171/16

RESUBMISSION

NOT RECOMMENDED: carfilzomib (Kyprolis®) is not recommended for use within NHS

Scotland in combination with lenalidomide and dexamethasone for the treatment of adult

patients with multiple myeloma who have received at least one prior therapy. Carfilzomib in combination with lenalidomide and dexamethasone improved progression free

survival compared with lenalidomide and dexamethasone in adults with relapsed and / or refractory multiple myeloma who had received one to three prior therapies.

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently ro b u st e co no mic

analysis to gain acceptance by SMC. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.

NOT RECOMMENDED



09 September 2020


22/97

Product

Manufacturer





Date of NHS Lothian

decision

carfilzomib 10mg, 30mg, 60mg powder

for solution for infusion (Kyprolis®)

Amgen Ltd.

07.08.17


Following a full submission assessed under the orphan process, carfilzomib (Kyprolis®) is

accepted for use within NHS Scotland. Indication under review: In combination with dexamethasone alone for the tre a tme n t o f a d u lt

patients with multiple myeloma who have received at least one prior therapy. Carfilzomib in combination with dexamethasone, compared with another proteasome inhibitor in

combination with dexamethasone, increased progression free survival in adults with relapsed o r refractory multiple myeloma who had received between one and three previous lines of

treatment. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost effectiveness of carfilzomib and is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.



guidance. Included on the Additional List, for Specialist Use only.

April 2018

cemiplimab 350mg concentrate for

solution for infusion (Libtayo®)

Sanofi

10.02.20


Following a full submission considered under the end of life and orphan equivalent process

cemiplimab (Libtayo®) is accepted for use within NHSScotland on an interim basis subject to

ongoing evaluation and future reassessment.

Indication under review: As monotherapy for the treatment of adult patients with me ta sta tic o r locally advanced cutaneous squamous cell carcinoma (CSCC) who are not candidates for

curative surgery or curative radiation. In a phase II study of cemiplimab in patients with metastatic or locally advanced CSCC the

objective response rate was 44%. The base-case economic analysis submitted by the company assumed that patients were

treated for a maximum of two years. This advice applies only in the context of an approved NHSScotland Patient Acce ss Sch eme




guidance. Included on the Additional List for Specialist Use only.

August 2020

ceritinib 150mg hard capsules (Zykadia®)

Novartis Europharm Limited

07.12.15 SMC Report No. 1097/15


ceritinib (Zykadia®) is accepted for use within NHS Scotland.

Indication under review: Treatment of adult patients with anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC) previously treated with crizotinib.

In two non-comparative studies (one phase I and one phase II) of patients with advanced AL K -positive NSCLC previously treated with crizotinib, treatment with ceritinib was associated with

clinically meaningful tumour responses and median overall survival of approximately 15 to 17 months. Controlled data with clinical outcomes are currently lacking.

This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e cost-effectiveness of ceritinib. This advice is contingent upon the continuing availa b il it y o f th e

PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.

Included on the Additional List, Specialist


April 2016

ceritinib 150mg hard capsules (Zykadia®)

Novartis Pharmaceuticals UK Ltd

09.04.18 SMC Report No. 1333/18

NON SUBMISSION

NOT RECOMMENDED: ceritinib (Zykadia®) is not recommended for use within NHS Scotland.

Indication under review: As monotherapy for the first-line treatment of adult patients with anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer.


NOT RECOMMENDED



09 September 2020


23/97

Product

Manufacturer





Date of NHS Lothian

decision

cetuximab 2mg/mLintravenousinfusion

(Erbitux®)

MerckKGaA

10.07.06


cetuximab (Erbitux®) is accepted for restricted use within NHS Scotland in combination with

radiation therapy for the treatment of patients with locally advanced squamous cell cancer of the head and neck.

It is restricted to patients who are not appropriate for or unable to tolerate chemo-radioth era p y and who are of good performance status with no evidence of distant metastases. It is also

restricted to use by specialists in the management of head and neck cancer.


Use only.

September 2006

cetuximab 5mg/mL solution for infusion (Erbitux

®)

Merck Serono

09.03.09 SMC Report No. 547/09

NON SUBMISSION

NOT RECOMMENDED: cetuximab (Erbitux®) is not recommended for use within NHS Scotla n d

for the treatment of patients with squamous cell cancer of the head and neck in combination with

platinum-based chemotherapy for recurrent and/or metastatic disease. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

cetuximab, 100mg/20mL and 500mg/100mL solution for infusion

(Erbitux®)

Merck Serono Ltd.

April 2017

NICE MTA 439 supersedes SMC Report No. 1012/14


(SMC Report No. 1012/14 superseded SMC Report No. 115/05 and SMC Report

No. 543/09)

NICE MTA 439 Cetuximab and panitumumab for previously untreated metastatic colorectal cancer

Cetuximab is recommended, within its marketing authorisation, as an option for previously untreated epidermal growth factor receptor (EGFR)-expressing, RAS wild-type metastatic

colorectal cancer in adults in combination with: 5-fluorouracil, folinic acid and oxaliplatin (FOLFOX), or

5-fluorouracil, folinic acid and irinotecan (FOLFIRI).

Includedon the Additional List, Specialist Use only.

May 2015

cinacalcet 30, 60 and 90 mg film-coated

tablets (Mimpara®)

Amgen Ltd

08.05.06


NOT RECOMMENDED: cinacalcet (Mimpara®) is not recommended for use within NHSScotland

for the reduction of hypercalcaemia in patients with parathyroid carcinoma. The h o ld e r o f th e marketing authorisation has not made a submission to SMC regarding this product in this

indication. As a result we cannot recommend its use within NHSScotland.

NOT RECOMMENDED

cisplatin/ vinorelbine


Adjuvant non-small cell lung cancer


Use only.

October 2005

cladribine 2mg/mL solution for injection

(LITAK®)

Lipomed GmbH

09.03.09



cladribine (Litak®) is accepted for use in NHS Scotland for the treatment of hairy cell leukaemia.

In patients for whom cladribine is an appropriate agent for this indication, the 2mg/mL so lu t io n allows administration by subcutaneous bolus injection over five consecutive days rather than b y

continuous intravenous infusion of the existing 1mg/mL solution for seven consecutive days. This may confer advantages in terms of convenience to patients and service delivery at a lo we r

cost per course.

New formulation of a product already

included in the Additional List.

March 2009



09 September 2020


24/97

Product

Manufacturer





Date of NHS Lothian

decision

clofarabine, 1mg/mL concentrate for

solution for infusion (Evoltra®)

Bioenvision Limited

15.01.07


clofarabine (Evoltra®) is accepted for restricted use within NHS Scotland for the treatment of

acute lymphoblastic leukaemia (ALL) in paediatric patients (= 21 years) who have re la p se d o r are refractory after receiving at least two prior regimens and where there is no other t re a tme n t

option anticipated to result in a durable response. It is restricted to patients in whom clofarabine is being used as a treatment to b rid g e to HSCT

and restricted to use by specialists in paediatric haemato-oncology. It is not cost effective when used for palliation.


Use only.

March 2009

cobimetinib (Cotellic®) 20mg film-coated

tablets Roche Products Ltd

12.09.16


NOT RECOMMENDED: cobimetinib (Cotellic®) is not recommended for use within NHS

Scotland in combination with vemurafenib for the treatment of adult patients with unresectable or metastatic melanoma with a BRAF V600 mutation.


NOT RECOMMENDED

crizotinib, 200mg and 250mg, hard

capsule (Xalkori®)

Pfizer Ltd.

07.10.13



crizotinib (Xalkori®) is accepted for use within NHS Scotland for the treatment of adults with

previously treated anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC).

In a phase III clinical study in patients with previously treated anaplastic lymphoma kinase (ALK)-positive advanced NSCLC, crizotinib significantly increased progression-free survival

compared with standard chemotherapy. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of crizotinib. This SMC advice is contingent upon the continuing availability of the patient access scheme in NHS Scotland or a list price that is equivalent or

lower.



November 2013

crizotinib, 200mg and 250mg hard

capsule (Xalkori®)

Pfizer Limited

11.07.16


crizotinib (Xalkori®) is accepted for use within NHS Scotland as first-line treatment of adults with

anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC). In patients with previously untreated advanced ALK-positive NSCLC, crizotinib significantly

improved progression-free survival compared with a standard systemic anti-cancer therapy. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of crizotinib. This advice is contingent upon the continuing availab il it y o f the PAS in NHS Scotland or a list price that is equivalent or lower.



October 2016

crizotinib, 200mg and 250mg hard

capsule (Xalkori®)

Pfizer Limited

04.05.18

SMC Report No 1329/18 Patient Access Scheme

Following a full submission assessed under the ultra-orphan medicine process.

crizotinib (Xalkori®) is accepted for use within NHS Scotland.

Indication under review: treatment of adults with ROS1-positive advanced non-sma ll ce ll lu n g

cancer (NSCLC). In a small, single arm, open-label, phase I study of patients with advanced ROS1-positive

NSCLC, treatment with crizotinib resulted in an objective response in 70% of patients. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of crizotinib. This advice is contingent upon the continuing availab il i t y o f the PAS in NHS Scotland or a list price that is equivalent or lower.




November 2018

cytarabine 50mg liposomal suspension for injection (DepoCyte

®)

Napp Pharmaceuticals

09.05.05 SMC Report No. 164/05

NOT RECOMMENDED: cytarabine liposomal suspension for injection (DepoCyte) is not recommended for use within NHS Scotland for the intrathecal treatment of lymphomatous

meningitis. Intrathecally administered cytarabine liposomal suspension cleared malignant cells from the

cerebrospinal fluid, however effects on symptom improvement were not well defined and the cost-effectiveness compared to cytarabine solution has not been demonstrated.

NOT RECOMMENDED



09 September 2020


25/97

Product

Manufacturer





Date of NHS Lothian

decision

dabrafenib, 50mg and 75mg hard

capsules (Tafinlar®)

GlaxoSmithKline

09.03.15


dabrafenib (Tafinlar®) is accepted for restricted use within NHS Scotland as monotherapy

treatment of adult patients with unresectable or metastatic melanoma with a BRAF V600 mutation.

SMC restriction: for use in patients with unresectable or metastatic BRAFV600 mutation-positive metastatic melanoma who have received no prior therapy.

In a phase III randomised open-label study, treatment with dabrafenib extended median progression free survival by 4.2 months compared with chemotherapy.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of dabrafenib. It is contingent upon the continuing availability of the

patient access scheme in NHS Scotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.



May 2015

dabrafenib 50mg and 75mg hard capsules (Tafinlar

®)


11.02.19 SMC Report No. 2131


dabrafenib (Tafinlar®) is accepted for use within NHSScotland.

Indication under review: In combination with trametinib for the adjuvant treatment of adult

patients with Stage III melanoma with a BRAF V600 mutation, following complete resection. Relapse-free survival was significantly longer in the dabrafenib plus trametinib group compa re d

with placebo in a phase III study of patients with completely resected, stage III mela n oma with BRAF V600E or V600K mutations.

This SMC advice takes account of the benefits of a Patient Access Schemes (PAS) that improves the cost-effectiveness of dabrafenib and trametinib.

Routinely available in line with national guidance. Included on the Additiona l L ist


May 2019

daclizumab 150mg/mL solution for

injection in prefilled syringe/pen (Zinbryta

®)

Biogen Idec Ltd.

10.04.17 SMC Report No. 1216/17


In adult patients for the treatment of relapsing forms of multiple sclerosis.

Restriction: for use

• in patients with rapidly evolving severe (RES) relapsing remitting multiple sclerosis (RRMS)

or

• in patients with RRMS with an inadequate response to disease modifying therapy




May 2017

WITHDRAWN FROM THE MARKET March 2018

dacomitinib 15mg, 30mg and 45mg

film-coated tablets (Vizimpro®)

Pfizer Ltd

09.09.19


dacomitinib (Vizimpro®) is accepted for use within NHSScotland.

Indication under review:as monotherapy, for the first-line treatment of adult patients with lo ca lly advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor

receptor (EGFR)-activating mutations. In an open-label, randomised, phase III study, dacomitinib significantly improved pro gre ssio n -

free survival compared with another EGFR tyrosine kinase inhibitor in adults with locally advanced or metastatic NSCLC with EGFR-activating mutations.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of dacomitinib. This advice is contingent upon the continuing availability of

the PAS in NHSScotland or a list price that is equivalent or lower.




October 2019

dalteparin (Fragmin®)

Pharmacia


Thromboprophylaxis in patients with oesophago-gastric cancer undergoing pre-operative cisplatin-based chemotherapy prior to surgery with curative intent.





December 2013



09 September 2020


26/97

Product

Manufacturer





Date of NHS Lothian

decision

daratumumab 20mg/mL concentrate for

solution for infusion (Darzalex®)

Janssen-Cilag Ltd

09.10.17



daratumumab (Darzalex®) is accepted for restricted use within NHS Scotland.

Indication under review: As monotherapy for the treatment of adult patients with re la p se d a n d refractory multiple myeloma, whose prior therapy included a proteasome inhibitor and an

immunomodulatory agent and who have demonstrated disease progression on the last therapy. SMC restriction: for use as a fourth line treatment option

In a pooled analysis of patients in a phase I/II and a phase II study, with heavily pre-treated multiple myeloma, who received the licensed dosing schedule of daratumumab, th e re wa s a n

overall response rate of 31%. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of daratumumab. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.




April 2018



Janssen-Cilag Ltd

13.05.19


NOT RECOMMENDED: daratumumab (Darzalex®) is not recommended for use within

NHSScotland. Indication under review: in combination with bortezomib, melphalan and prednisone for the

treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant.


NOT RECOMMENDED



Janssen-Cilag Ltd

08.07.19


daratumumab (Darzalex®) is accepted for restricted use within NHSScotland.

Indication under review: In combination with lenalidomide and dexamethasone, or b o rte zo mib and dexamethasone, for the treatment of adult patients with multiple myeloma who have

received at least one prior therapy. SMC restriction: in combination with bortezomib and dexamethasone, for the treatment of a d ult

patients with multiple myeloma who have received one prior therapy only. Progression-free survival was significantly longer in patients who received daratumumab in

combination with bortezomib and dexamethasone compared with those who received bortezomib and dexamethasone in a phase III study in patients with multiple myeloma who h a d

received at least one prior therapy. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of daratumumab. This advice is contingent upon the continuing availability of the PAS in NHSScotland or a list price that is equivalent or lower.




November 2019

daratumumab 20mg/mL concentrate for solution for infusion (Darzalex

®)

Janssen-Cilag Ltd

10.02.20 SMC Report No. 2269

NON SUBMISSION

NOT RECOMMENDED: daratumumab (Darzalex®) is not recommended for use within

NHSScotland.

Indication under review: In combination with lenalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for

autologous stem cell transplant. The holder of the marketing authorisation has not made a submission to SMC regarding

this product in this setting. As a result we cannot recommend its use within NHSScotland.

NOT RECOMMENDED

darbepoetin alfa (Aranesp®)

Amgen Ltd

November 2014


NICE MTA 323 Erythropoiesis-stimulating agents for treating anaemia in people with cancer

having chemotherapy erythropoiesis-stimulating agents (epoetin alfa, beta, theta and zeta, and darbepoetin alfa) are

recommended, within their marketing authorisations, as options for treating anaemia in people with cancer who are having chemotherapy.

If different erythropoiesis-stimulating agents are equally suitable, the product with the lowest acquisition cost for the course of treatment should be used.

Not routinely available there is a local preference for alternative medicines.

December 2014



09 September 2020


27/97

Product

Manufacturer





Date of NHS Lothian

decision

dasatinib 20mg, 50mg, 80mg, 100mg and

140mg film-coated tablets (Sprycel®)

Bristol-Myers Squibb Pharmaceuticals Ltd

12.09.16



dasatinib (Sprycel®) is accepted for use within NHS Scotland for the treatment of adult patie n ts

with chronic, accelerated or blast phase chronic myelogenous leukaemia (CML) with resista n ce or intolerance to prior therapy including imatinib mesilate.

In patients with chronic, accelerated or blast phase CML, dasatinib produced h ae ma tolo g ical and cytogenetic responses in two phase III dosing ranging studies. In a phase II study dasatinib

was associated with higher haematological and cytogenetic responses relative to another tyrosine kinase inhibitor in patients with chronic phase CML.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of dasatinib. This advice is contingent upon the continuing availabil it y o f

the PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.



December 2016

dasatinib, 20mg, 50mg, 70mg tablets (Sprycel

®)


07.05.07 SMC Report No. 371/07

NOT RECOMMENDED: dasatinib 20mg, 50mg, 70mg (Sprycel®) is not recommended for use

within NHS Scotland for the treatment of adults with Philadelphia chromosome positive (Ph+)

acute lymphoblastic leukaemia with resistance or intolerance to prior therapy. It has been associated with haematological and cytogenetic responses in patients re sista n t o r

intolerant to existing treatment. However, the economic case was not sufficiently robust and the manufacturer’s justification of the treatment’s cost in relation to its health benefits was not

sufficient to gain acceptance by SMC.

NOT RECOMMENDED

dasatinib 20mg, 50mg, 80mg, 100mg and 140mg film-coated tablets (Sprycel

®)


12.09.16 SMC Report No. 1170/16


dasatinib (Sprycel®) is accepted for use within NHS Scotland for the treatment of adult patie n ts

with newly diagnosed Philadelphia chromosome positive (Ph+) chronic myelogenous leukaemia

(CML) in the chronic phase. In an open-label, phase III study, dasatinib was associated with significantly higher cytoge ne t ic

and molecular response rates at 12 months compared with another tyrosine kinase inhibitor. There were no differences in progression-free or overall survival.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of dasatinib. This advice is contingent upon the continuing availabil it y o f


Includedon the Additional List, for Specialist Use only, for the indication in

question.

December 2016

dasatinib20mg, 50mg, 80mg, 100mg, 140

mg film-coated tablets (Sprycel) Bristol-Myers Squibb Pharmaceuticals Ltd

08.04.19

SMC report No. 2142 PRODUCT UPDATE


dasatinib (Sprycel®) is accepted for use within NHSScotland for the treatment of paediatric

patients with newly diagnosed Philadelphia chromosome positive chronic myelogenous leukaemiain chronic phase (Ph+ CML-CP) or Ph+ CML-CP resistant or intolerant to prior therapy

including imatinib. SMC has previously accepted dasatinib for use in the treatment of adult patients with newly

diagnosed Ph+ CML-CP (SMC No. 1170/16) and Ph+ CML-CP resistant or intolerant to prior therapy including imatinib (SMC No. 370/07).

Dasatinib was accepted for use in the treatment of adult patients with Ph+ CML-CP resista n t o r intolerant to prior therapy including imatinib (SMC No. 370/07) following a submission under th e

orphan process. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of dasatinib. This SMC advice is contingent upon the continuing availability of the patient access scheme or a list price that is equivalent or lower.


guidance. Included on the Additiona l L ist for Specialist Use only

April 2019

dasatinib, 20mg, 50mg, 80mg, 100mg

and 140mg film-coated tablets (Sprycel®)


13.05.19


NOT RECOMMENDED: dasatinb (Sprycel®) is not recommended for use within NHSScotland.

Indication under review: the treatment of paediatric patients with newly diagnosed Philad elp h ia chromosome positive acute lymphoblastic leukaemia in combination with chemotherapy.


NOT RECOMMENDED



09 September 2020


28/97

Product

Manufacturer





Date of NHS Lothian

decision

decitabine (Dacogen®) 50 mg powder fo r

concentrate for solution for infusion Janssen-Cilag Ltd

14.01.13


NOT RECOMMENDED: decitabine (Dacogen®) is not recommended for use within NHS

Scotland for the treatment of adult patients aged 65 years and above with newly diagn o sed d e novo or secondary acute myeloid leukaemia (AML), according to the World Health Organisation

(WHO) classification, who are not candidates for standard induction chemotherapy. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

defibrotide, 80mg/mL, concentrate for

solution for infusion (Defitelio®)

Gentium GmbH

09.06.14


defibrotide (Defitelio®) is accepted for use within NHS Scotland for the treatment of severe

hepatic veno-occlusive disease (VOD) also known as sinusoidal obstruction syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy.

In a phase III open-label study, defibrotide was associated with improved comp le te re sp o n se rate and survival in patients with severe VOD, compared with a historical control group.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of defibrotide. This SMC advice is contingent upon the continuing




August2014

degarelix 120mg, 80mg powder and

solvent for solution for injection (Firmagon

®)

Ferring Pharmaceuticals Ltd

17.01.11 SMC Report No. 560/09


degarelix (Firmagon®) is accepted for use within NHS Scotland. Degarelix is a gonadotropin -

releasing hormone (GnRH) antagonist indicated for the treatment of adult male patients with advanced hormone-dependent prostate cancer.

In one study that included patients with all stages of prostate cancer, degarelix was shown to be non-inferior to a luteinising hormone releasing hormone (LHRH) agonist in suppressing

testosterone levels over a one year treatment period without an initial testosterone flare. This SMC advice takes account of the benefits of a patient access scheme (PAS) that improve s

the cost-effectiveness of degarelix. This SMC advice is contingent upon the continuing availability of the patient access scheme in NHS Scotland.

Included on the Additional List for

Specialist Use only and a prescribing note, for use in patients who are at risk of spinal

cord compression.

Routinely available in line with national guidance. Included on the Additional List,

for Specialist Initiation.

August 2012

August 2018

denosumab 60mg solution for injection in pre-filled syringe

(Prolia®)

Amgen Ltd

13.12.10


NOT RECOMMENDED: denosumab (Prolia®) is not recommended for use within NHS Scotland.

Indication under review: bone loss associated with hormone ablation in men with prostate

cancer at increased risk of fractures. The holder of the marketing authorisation has not made a submission to SMC regarding this

product in this indication. As a result we cannot recommend its use with in NHS Scotland.

NOT RECOMMENDED

denosumab 120 mg solution for injectio n

(Xgeva®)

Amgen

October 2012


denosumab(Xgeva®)is recommended as an option for preventing skeletal-related events

(pathological fracture, radiation to bone, spinal cord compression or surgery to bone) in a d u lts with bone metastases from breast cancer and from solid tumours other than prostate if:

• bisphosphonates would otherwise be prescribed, and

• the manufacturer provides denosumab with the discount agreed in the patient acce ss scheme.

Included in the LJF, for Specialist Use

only, for the indication in question. Application was for breast cancer patients

only.

April 2013

denosumab (Xgeva®) 120mg solution for

injection Amgen Ltd

07.12.15


NOT RECOMMENDED: denosumab (Xgeva®) is not recommended for use within NHS

Scotland. Indication under review:Adults and skeletally mature adolescents with giant cell tumour of bone

that is unresectable or where surgical resection is likely to result in severe morbidity The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED



09 September 2020


29/97

Product

Manufacturer





Date of NHS Lothian

decision

denosumab (Xgeva)®) 120mg solution for

injection Amgen Ltd

13.08.18


NOT RECOMMENDED: denosumab (Xgeva®) is not recommended for use within NHSScotland.

Indication under review: Prevention of skeletal related events (pathological fracture, radiation to bone, spinal cord compression or surgery to bone) in adults with haematological malignancies

involving bone. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

dexamethasone 2mg and 4mg

soluble tablets


Inflammation, various.

The 2mg and 4mg soluble tablets are more cost effective than the non-soluble tablets a n d th e 4mg tablet may reduce the tablet burden in patients receiving large dexamethasone doses.

Soluble tablets are also advantageous for patients with swallowing difficulties.

Routinely available in line with local

guidance. Included in the LJF.

August 2017

dexamethasone 40mg tablets (Neofordex

®)

Aspire Pharma Ltd

12.03.18 SMC Report No. 1322/18

NON SUBMISSION

NOT RECOMMENDED: dexamethasone (Neofordex®) is not recommended for use within NHS

Scotland.

Indication under review: In adults for the treatment of symptomatic multiple myeloma in combination with other medicinal products


NOT RECOMMENDED

dexrazoxane (Cardioxane®)


12.11.07 SMC Report No. 419/07

NON SUBMISSION

NOT RECOMMENDED: dexrazoxane (Cardioxane®) is not recommended for use within NHS

Scotland for the prevention of chronic cumulative cardiotoxicity caused by doxorubicin or epirubicin use in advanced and/or metastatic cancer patients after previous anthracycline

containing treatment. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

dexrazoxane 20mg/mL, for infusion (Savene

®)

TopoTarget A/S

13.10.08 SMC Report No. 361/07

RESUBMISSION

NOT RECOMMENDED: dexrazoxane (Savene®) is not recommended for use within NHS

Scotland for the treatment of anthracycline extravasation. Data from non-comparative, open-label phase II/III studies indicate that administration of dexrazoxane is associated with a relatively low rate of surgery and adverse sequelae follo win g

extravasation of anthracyclines. The manufacturer did not present a sufficiently robust economic analysis to gain acceptance b y

SMC and in addition the justification of the treatment’s cost in relation to its health benefit s wa s not sufficient.

NOT RECOMMENDED



09 September 2020


30/97

Product

Manufacturer





Date of NHS Lothian

decision

dinutuximab beta 4.5mg/mL concent ra te

for solution for infusion (Qarziba®)

Eusa Pharma

12.11.18

SMC Report No 2105 Patient Access Scheme

Following a full submission assessed under the ultra-orphan process, dinutuximab beta

(Qarziba®) is accepted for use within NHSScotland.

Indication under review: for the treatment of high-risk neuroblastoma in patients aged 12 months

and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as

patients with history of relapsed or refractory neuroblastoma, with or without residua l d ise a se . Prior to the treatment of relapsed neuroblastoma, any actively progressing disea se sh o u ld b e

stabilised by other suitable measures. In patients with a history of relapsed/refractory disease and in patients who have not achieved a

complete response after first line therapy, dinutuximab beta should be combined with interleukin-2.

Comparisons with historical controls indicate that dinutuximab beta plus isotretinoin with and without aldesleukin (interleukin-2) improved event-free survival and overall survival in p a t ie n ts

undergoing first-line treatment for high-risk neuroblastoma and improved overall survival in patients with relapsed neuroblastoma. In patients with relapsed or refractory neuroblastoma

dinutuximab beta in combination with isotretinoin and aldesleukin was associated with tu mo u r responses.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of dinutuximab beta. This advice is contingent upon the continuing

availability of the PAS in NHSScotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.

Routinelyavailable in line with national


December 2018

docetaxel (Taxotere) Aventis

09.05.03


docetaxel, in combination with cisplatin, is an effective treatment option for the first line treatment of unresectable, locally advanced or metastatic (stage III/IV) non -small cell lung

cancer (NSCLC). In common with the other drugs recommended by Quality Improvement Scotland (QIS) fo r th is

condition, benefit has only been proven in patients with good performance status. Est ima te d cost per quality adjusted life year (QALY) gained is relatively high. Docetaxel should be initiated

by respiratory physicians/oncologists experienced in the treatment of NSCLC.

Included in Formulary.

Taxotere brand has been discontinued. In NHS Lothian generic docetaxel is used for

this indication.

June 2019

docetaxel (Taxotere®) injection

concentrate

Sanofi-Aventis UK

13.11.06 SMC Report No. 333/06

NON SUBMISSION

NOT RECOMMENDED: docetaxel (Taxotere®) injection concentrate in combination with

cisplatin and 5-fluorouracil is not recommended for use within NHS Scotland for the treatment of

patients with metastatic gastric adenocarcinoma, including adenocarcinoma of the gastroesophageal junction, who have not received prior chemotherapy for metastatic disease.


NOT RECOMMENDED

docetaxel 20 and 80mg concentrate a n d

solvent for solution for infusion (Taxotere

®)

Sanofi-Aventis

07.05.07 SMC Report No. 369/07

docetaxel (Taxotere®) is accepted for restricted use within NHS Scotland for the induction

treatment of patients with unresectable locally advanced squamous cell carcinoma of th e h e a d and neck in combination with cisplatin and 5-fluorouracil. It is restricted to patients in whom

induction chemotherapy is appropriate. The docetaxel-containing induction regimen was associated with improved progression-free and

overall survival, compared with cisplatin and 5-fluorouracil alone, in patients with good performance status.


Use only.


this indication.

July 2007

June 2019



09 September 2020


31/97

Product

Manufacturer





Date of NHS Lothian

decision

docetaxel 20mg, 80mg concentrate and

solvent for solution for infusion, single dose vials (Taxotere

®)

Sanofi-Aventis

10.10.05 SMC Report No. 201/05

docetaxel (Taxotere®) is accepted for use within NHS Scotland in combination with doxorub ic in

and cyclophosphamide for the adjuvant treatment of operable, node-positive breast cancer. Docetaxel in combination with doxorubicin and cyclophosphamide was associated with a

significant improvement in disease free survival at 5 years when compared with one of the standard treatment regimens. However, this benefit is associated with an increased risk of

toxicity. Docetaxel has demonstrated cost effectiveness in comparison to standard t re a tme n t regimen used in NHS Scotland.

Added to the Formulary, for Specialist Use

only.


this indication.

November 2005

June 2019

docetaxel concentrate and solvent for

solution for infusion, single dose vials (Taxotere

®)

Sanofi-Aventis

June 2006 NICE MTA 101 supersedes SMC Report

No. 209/05

NICE MTA 101 Docetaxel for the treatment of hormone-refractory metastatic prostate cancer.

Docetaxel is recommended, within its licensed indications, as a treatment option fo r me n with hormone-refractory metastatic prostate cancer only if their Karnofsky performance-status sco re

is 60% or more. It is recommended that treatment with docetaxel should be stopped: •at the completion of planned treatment of up to 10 cycles, or

•if severe adverse events occur, or •in the presence of progression of disease as evidenced by clinical or laboratory crite ria , o r b y

imaging studies. Repeat cycles of treatment with docetaxel are not recommended if the disease recurs after

completion of the planned course of chemotherapy.

Added to the LJF as first choice, for



this indication.

January 2007

June 2019

docetaxel 20 and 80mg concentrate a n d solvent for solution for infusion

(Taxotere®)

Sanofi-aventis

07.07.08


docetaxel (Taxotere®) is accepted for restricted use within NHS Scotland for the induction

treatment of patients with resectable locally advanced squamous cell carcinoma of the head and

neck in combination with cisplatin and 5-fluorouracil. It is restricted to patients in whom induction chemotherapy is appropriate. In the pivo ta l stu d y,

which included patients with technically resectable disease, the docetaxel-containing in d uct io n regimen was associated with improved overall survival compared with cisplatin and 5 -

fluorouracil alone. SMC has previously issued advice for patients with unresectable disease and this now exte n ds

the advice to patients with resectable disease.

Added to the Additional List, Specialist Use only.

Taxotere brand has been discontinued. In

NHS Lothian generic docetaxel is used for this indication.

May 2009

June 2019

docetaxel (Taxotere®) 20 mg/1mL and 80

mg/4mL and 160 mg/8mL concentrate for solution for infusion

Sanofi Aventis

08.11.10 SMC Report No. 659/10

NON SUBMISSION

NOT RECOMMENDED: docetaxel (Taxotere®) in combination with doxorubicin and

cyclophosphamide is not recommended for use within NHS Scotland. Indication under review: adjuvant treatment of patients with operable node-negative breast

cancer. The holder of the marketing authorisation has not made a submission to SMC regarding this

product in this indication. As a result SMC cannot recommend its use within NHS Scotland.

NOT RECOMMENDED

docetaxel


Treatment in patients with newly diagnosed (within 3 months of starting LHRH analogue or antagonist therapy) metastatic or locally advanced

(TanyN1M0 or T3-4N0M0) prostate cancer with a performance status of 0 -1 and no contraindication to chemotherapy (e.g. marrow failure).

Added on the Additional List, for Specialist Use only.




In NHS Lothian generic docetaxel is u se d for this indication.

September 2015

June 2019



09 September 2020


32/97

Product

Manufacturer





Date of NHS Lothian

decision

docetaxel, oxaliplatin, folinic acid, 5-

fluorouracil (FLOT)

Local Formulary process

Peri-operative chemotherapy for oesophago-gastric adenocarcinoma.

Included on the Additional List for

Specialist Use Only.

docetaxel, oxaliplatin, folinic acid, 5-fluorouracil (FLOT) for peri-operative

chemotherapy for oesophago-gastric adenocarcinoma has been classified RED

under the ADTC “Policy for the use of unlicensed (and off-label use) Medicines in

NHS Lothian.

July 2019

durvalumab 50mg/mL concentrate for

solution for infusion (Imfinzi®)

AstraZeneca

10.06.19 SMC Report No. 2156


durvalumab (Imfinzi®) is accepted for use within NHSScotland.

Indication under review: as monotherapy for the treatment of locally advanced, unresectable non-small cell lung cancer (NSCLC) in adults whose tumours express PD-L1 [programme d ce ll

death ligand 1] on ≥1% of tumour cells and whose disease has not progressed following platinum-based chemoradiation therapy.

Durvalumab, compared with placebo, improved progression-free survival and overall surviva l in adults who have locally advanced unresectable NSCLC with PD-L1 expressed on ≥1% of

tumour cells and disease that has not progressed following platinum-based chemoradiation therapy.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of durvalumab. This advice is contingent upon the continuing availa bil it y

of the PAS in NHSScotland or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.



July 2019

edoxabantosilate 15mg, 30mg, 60mg

film-coated tablets (Lixiana®) Daiichi Sankyo UK Limited

09.10.15


edoxaban (Lixiana®) is accepted for use within NHS Scotland.

Indication under review: Treatment of deep vein thrombosis (DVT) and pulmonary embolism (PE), and prevention of recurrent DVT and PE in adults in malignant disease.

One phase III study showed non-inferiority of edoxaban versus a vitamin K antagonist for venous thromboembolism recurrence in patients who had received at least five days tre a tme nt

with low molecular weight heparin or unfractionated heparin. Edoxaban was also associated with a significant reduction in the risk of major and clinically relevant non-major bleeding (compo site

endpoint).



January 2019

elotuzumab (Empliciti®) 300mg and

400mg powder for concentrate for solution for infusion

Bristol Myers Squibb Pharmaceutical Limited

08.08.16


NOT RECOMMENDED: elotuzumab (Empliciti®) is not recommended for use within NHS

Scotland for the treatment of multiple myeloma in combination with lenalidomide and dexamethasone in adult patients who have received at least one prior therapy.


NOT RECOMMENDED



09 September 2020


33/97

Product

Manufacturer





Date of NHS Lothian

decision

eltrombopag (Revolade®) film-coated

tablets 25mg and 50mg Novartis UK Ltd

16.01.17



eltrombopag (Revolade®) is accepted for restricted use within NHS Scotland for chronic immune

(idiopathic) thrombocytopenic purpura (ITP) patients aged 1 year to 17 years who are refracto ry to other treatments (e.g. corticosteroids, immunoglobulins).

SMC restriction: use in patients with severe symptomatic ITP or a high risk of bleeding. Eltrombopag has previously been accepted for restricted use in adult patients with chronic

immune (idiopathic) thrombocytopenic purpura. The license has been extended to include children from 1 year.

SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves th e cost-effectiveness of eltrombopag. This advice is contingent upon the continuing availa bil it y o f




January 2017

encorafenib 50mg and 75mg hard capsules (Braftovi

®)

Pierre Fabre Ltd

08.07.19 SMC Report No. 2145

NOT RECOMMENDED: encorafenib (Braftovi®)is not recommended for use within

NHSScotland.

Indication under review: In combination with binimetinib for the treatment of adult patien ts with unresectable or metastatic melanoma with a BRAF V600 mutation.

Progression-free survival was significantly longer in the encorafenib plus binimetinib group compared with BRAF inhibitor monotherapy in a phase III study of patients with unresectable o r

metastatic BRAF V600 melanoma. The submitting company did not present a sufficiently robust economic analysis to gain

acceptance by SMC. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.

NOT RECOMMENDED

encorafenib 50mg and 75mg hard

capsules (Braftovi®)

Pierre Fabre Ltd

10.02.20 SMC Report No. 2238


Following a resubmission assessed under the end of life and orphan medicine process

encorafenib (Braftovi®) is accepted for use within NHSScotland

Indication under review: In combination with binimetinib for the treatment of adult patie nts with

unresectable or metastatic melanoma with a BRAF V600 mutation. Progression-free survival was significantly longer in the encorafenib plus binimetinib group

compared with BRAF inhibitor monotherapy in a phase III study of patients with unresectable o r metastatic BRAF V600 melanoma.

This advice applies only in the context of approved NHSScotland Patient Access Scheme (PAS) arrangements delivering the cost-effectiveness results upon which the decision was b a se d, o r

PAS/ list prices that are equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.


guidance. Included on the Additional List for Specialist Use only.

July 2020

enzalutamide 40mg soft capsules (Xtandi

®)

Astellas Pharma Ltd

11.11.13 SMC Report No. 911/13


enzalutamide (Xtandi®) is accepted for use within NHS Scotland for the treatment of ad ult me n

with metastatic castration-resistant prostate cancer (mCRPC) whose disease has progressed on

or after docetaxel therapy. In one randomised, double-blind, phase III clinical study, enzalutamide significantly in cre a sed

overall survival compared with placebo. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of enzalutamide. This SMC advice is contingent upon the continuing availability of the patient access scheme in NHS Scotland or a list price that is equivalent or

lower.


question.

December 2013



09 September 2020


34/97

Product

Manufacturer





Date of NHS Lothian

decision

enzalutamide, 40mg soft capsules

(Xtandi®)

Astellas Pharma Ltd

07.03.16

SMC Report No. 1066/15 INDEPENDENT REVIEW PANEL


enzalutamide (Xtandi®) is accepted for use within NHS Scotland.

Indication under review: Treatment of adult men with metastatic castration-resistant prostate cancer (mCRPC) who are asymptomatic or mildly symptomatic after failure of androgen

deprivation therapy in whom chemotherapy is not yet clinically indicated. In a randomised, double-blind phase III study of adult men with chemothera py n a ive mCRPC

treatment with enzalutamide was associated with a statistically significant extended overall survival and radiographic progression free survival compared to placebo.

This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e cost-effectiveness of enzalutamide. This advice is contingent upon the continuing availabilit y o f

the patient access scheme in NHS Scotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.



April 2016

enzalutamide 40mg soft capsules (Xtandi

®)

Astellas Pharma Ltd

07.10.19 SMC Report No. 2195

NOT RECOMMENDED: following a full submission considered under the orphan equivalent process, enzalutamide (Xtandi

®) is not recommended for use within NHSScotland.

Indication under review: The treatment of adult men with high-risk non-metastatic castration-resistant prostate cancer (CRPC).

In a phase III study in men with high-risk non-metastatic CRPC enzalutamide was superior to placebo for metastasis-free survival. High-risk was defined as prostate specific antigen (PSA)

doubling time ≤10 months and PSA ≥2 nanograms/mL. Both groups received on -going androgen-deprivation therapy or had undergone bilateral orchiectomy. Overall survival data a re

immature. The submitting company’s justification of the treatment’s cost in relation to its health benefits

was not sufficient and in addition the company did not present a sufficiently ro b u st e co no mic analysis to gain acceptance by SMC.


NOT RECOMMENDED

epirubicin (Pharmorubicin®), cisplatin

(Platinex®) and capecitabine (Xeloda

®)

(ECX)

Pharmacia, Bristol-Myers Squibb, Roche


Peri-operative treatment of operable gastric and type 3 oesophagogastric junctional

adenocarcinomas.

Added to the LJF as first choice treatme n t

for peri-operative treatment of operable gastric and type 3 oesophagogastric

junctional adenocarcinomas, Specialist Use only.

September 2007



09 September 2020


35/97

Product

Manufacturer





Date of NHS Lothian

decision

eribulin (mesilate) 0.44mg/mLsolution for

injection (Halaven®)

Eisai Ltd

07.03.16

SMC Report No. 1065/15 (Supersedes SMC Report No. 726/11)


eribulin (Halaven®) is accepted for restricted use within NHS Scotland.

Indication under review: for the treatment of adult patients with locally advanced or me ta sta t ic breast cancer who have progressed after at least one chemotherapeutic regimen for adva n ced

disease. Prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting unless patients were not suitable for these treatments.

SMC restriction: for use in patients with locallyadvanced or metastatic breast cancer wh o h a ve progressive disease after at least two prior chemotherapeutic regimens for advan ced d ise ase

which includes capecitabine if indicated. In a randomised, phase III, open-label study, median overall survival was extended by 2.5

months in patients treated with eribulin compared with the comparator, treatment of physicia n ’s choice, which included a range of single agent chemotherapy treatments. In th e su b g ro up o f

patients previously treated with capecitabine the extension to median overall surviva l wa s 2 .9 months.

This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e cost-effectiveness of eribulin. This advice is contingent upon the continuing availa b il it y o f th e

PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.



July 2016

eribulin 0.44mg/mL solution for injection (Halaven

®)

Eisai Ltd

09.09.19 SMC Report No. 2231

NON SUBMISSION

NOT RECOMMENDED: in the absence of a submission from the holder of the marketing authorisation eribulin (Halaven

®) is not recommended for use within NHSScotland for the

treatment of adult patients with unresectable liposarcoma who have received prior anthracycline containing therapy (unless unsuitable) for advanced or metastatic d isease.


NOT RECOMMENDED

erlotinib (Tarceva®)


09.07.07 SMC Report No. 382/07

NON SUBMISSION

NOT RECOMMENDED: erlotinib (Tarceva®) in combination with gemcitabine is not

recommended for use within NHS Scotland for the treatment of patients with metastatic pancreatic cancer.


NOT RECOMMENDED



09 September 2020


36/97

Product

Manufacturer





Date of NHS Lothian

decision

erlotinib, 100 and 150mg film-coated

tablets (Tarceva®)

Roche

December 2015



NICE MTA374 Erlotinib and gefitinib for treating non-small-cell lung cancer that has progressed

after prior chemotherapy Erlotinib is recommended as an option for treating locally advanced or metastatic non-small-ce ll

lung cancer that has progressed in people who have had non-targeted chemotherapy be cau se of delayed confirmation that their tumour is epidermal growth factor receptor tyrosine kinase

(EGFR-TK) mutation-positive, only if the company provides erlotinib with the discount agreed in the patient access scheme revised in the context of NICE technology appraisal guidance 258.

Erlotinib is recommended as an option for treating locally advanced or metastatic non-small-ce ll lung cancer that has progressed after non-targeted chemotherapy in people with tumours of

unknown EGFR-TK mutation status, only if:

• the result of an EGFR-TK mutation diagnostic test is unobtainable because of an inadequate tissue sample or poor-quality DNA and

• the treating clinician considers that the tumour is very likely to be EGFR-TK mutatio n -positive and

• the person’s disease responds to the first 2 cycles of treatment with erlotinib and

• the company provides erlotinib with the discount agreed in the patient access scheme revised in the context of NICE technology appraisal guidance 258.

Erlotinib is not recommended for treating locally advanced or metastatic non-small-cell lung

cancer that has progressed after non-targeted chemotherapy in people with tu mo u rs th a t a re EGFR-TK mutation-negative.

Gefitinib is not recommended for treating locally advanced or metastatic non-small-cell lung cancer that has progressed after non-targeted chemotherapy in people with tu mo u rs th a t a re

EGFR-TK mutation-positive.


Use only.

September 2006

erlotinib, 25, 100 and 150mg film-co a te d

tablets (Tarceva®)

Roche

17.01.11


NOT RECOMMENDED: erlotinib (Tarceva®) is not recommended for use within NHS Scotland.

Indication under review: as monotherapy for maintenance treatment in patients with locally advanced or metastatic non-small cell lung cancer with stable disease after 4 cycles of standard

platinum-based first-line chemotherapy. Erlotinib maintenance treatment provided a statistically significant increase in progressio n f re e

survival and overall survival in patients treated with standard first-line platinum-based chemotherapy, both in the whole study population and in a post hoc analysis in p a t ie n ts with

stable disease. In the whole study population the changes in these outcomes were consid e re d to be of modest size.

The manufacturer’s justification of the treatment’s cost in relation to its health benefit s wa s n o t sufficient to gain acceptance by SMC.

NOT RECOMMENDED

erlotinib 25, 100 and 150mg film-coated

tablets (Tarceva®)

Roche Products Ltd

16.01.12


erlotinib (Tarceva®) is accepted for use within NHS Scotland for first-line treatment of patients

with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epiderma l g ro wth factor receptor (EGFR) activating mutations.

In patients with advanced or metastatic NSCLC with EGFR mutations, erlotinib was associa te d with significantly improved progression-free survival compared with platinum-based doublet

chemotherapy regimens. There are no mature overall survival data. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of erlotinib. This SMC advice is contingent upon the continuing availability of the PAS in NHS Scotland.


Use only.

March 2012



09 September 2020


37/97

Product

Manufacturer





Date of NHS Lothian

decision

etanercept (Enbrel®)


Immunosuppressant therapy for islet cell transplant.

Routunely available in line with regional

guidance. Included on the Additional L ist , for Specialist Use only



March 2019

everolimus 2.5mg, 5mg and 10mg tablets

(Afinitor®)

Novartis Pharmaceuticals UK Limited

10.11.14


everolimus (Afinitor®) is accepted for use within NHS Scotland for the treatment of patients with

advanced renal cell carcinoma, whose disease has progressed on or after treatment with vascular endothelial growth factor (VEGF)-targeted therapy.

Everolimus, in conjunction with best supportive care (BSC), increased median progression-f re e survival (PFS) by three months compared with placebo plus BSC in heavily pre-treated patien ts

with metastatic renal cell carcinoma.



December 2014

everolimus (Votubia®) 2.5mg and 5mg

tablets Novartis Pharmaceuticals UK Ltd

09.04.12

SMC Report No. 787/12 NONSUBMISSION

NOT RECOMMENDED: everolimus (Votubia®) is not recommended for use within NHS

Scotland for the treatment of patients aged 3 years and older with subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC) who require therapeutic

intervention but are not amenable to surgery. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

everolimus, 5mg, 10mg tablets (Afinitor®)


July 2017



everolimus (Afinitor®) is accepted for use within NHS Scotland for the Treatment of unresectable

or metastatic, well- or moderately-differentiated neuroendocrine tumours of pancreatic origin

(pNET) in adults with progressive disease. Everolimus was superior to placebo in prolonging progression-free survival in adults with

progressive, advanced pNET who were receiving best supportive care. NICE MTA 449Everolimus and sunitinib for treating unresectable or metastatic neuroendocrin e

tumours in people with progressive disease. Everolimus and sunitinib are recommended, within their marketing authorisations, as options for

treating well- or moderately-differentiated unresectable or metastatic neuroendocrin e tu mo u rs (NETs) of pancreatic origin in adults with progressive disease.

Everolimus is recommended, within its marketing authorisation, as an option for treating well-differentiated (grade 1 or grade 2) non-functional unresectable or metastatic NETs of

gastrointestinal or lung origin in adults with progressive disease.

Includedon the Additional List, for Specialist Use only.

August 2017

everolimus (Votubia®) 10mg tablets

Novartis Pharmaceuticals Ltd

10.06.13


NOT RECOMMENDED: everolimus (Votubia®) is not recommended for use within NHS

Scotland for the treatment of adult patients with renal angiomyolipoma associated with tuberous

sclerosis complex (TSC) who are at risk of complications (based on factors such as tumour size or presence of aneurysm, or presence of multiple or bilateral tumours) but who do n o t re q u ire

immediate surgery. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED



09 September 2020


38/97

Product

Manufacturer





Date of NHS Lothian

decision


(Afinitor®)


11.04.16


nd RESUBMISSION

everolimus (Afinitor®) is accepted for use within NHS Scotland.

Indication under review: For the treatment of hormone receptor-positive, HER2/neu negative advanced breast cancer, in combination with exemestane, in postmenopausal women without

symptomatic visceral disease after recurrence or progression following a non-steroidal aromatase inhibitor.

The addition of everolimus to exemestane treatment significantly increased progression free survival compared with exemestane alone in postmenopausal women with disease progression

following a non-steroidal aromatase inhibitor. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of everolimus. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.




July 2016

everolimus (Certican®) 0.25mg, 0.5mg

and 0.75mg tablets Novartis Pharmaceuticals UK Ltd

09.11.15


NOT RECOMMENDED: everolimus (Certican®) is not recommended for use within NHS

Scotland. Indication under review:Prophylaxis of organ rejection in adult patients at low to moderate

immunological risk receiving a cardiac transplant Prophylaxis of organ rejection in patients receiving a hepatic transplant


NOT RECOMMENDED


(Afinitor®)


July 2017



NICE MTA 449Everolimus and sunitinib for treating unresectable or metastatic neuroendocrin e

tumours in people with progressive disease. Everolimus and sunitinib are recommended, within their marketing authorisations, as options for

treating well- or moderately-differentiated unresectable or metastatic neuroendocrin e tu mo u rs (NETs) of pancreatic origin in adults with progressive disease.

Everolimus is recommended, within its marketing authorisation, as an option for treating well-differentiated (grade 1 or grade 2) non-functional unresectable or metastatic NETs of

gastrointestinal or lung origin in adults with progressive disease.


guidance. Included on the Additional L ist , Specialist Use only, for the indication in

question.

Augist 2017

everolimus 0.25mg, 0.5mg and 0.75mg

tablets (Certican®)


13.11.17


NOT RECOMMENDED: everolimus (Certican®) is not recommended for use within NHS

Scotland. Indication under review: Prophylaxis of organ rejection in adult patients at low to moderate

immunological risk receiving an allogenic renal transplant. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

exemestane 25mg tablets (Aromasin®)

Pfizer Limited

07.11.05 SMC Report No. 210/05

exemestane (Aromasin®) is accepted for restricted use within NHS Scotland for the adjuvant

treatment of postmenopausal women with oestrogen receptor positive invasive early breast cancer, following 2 - 3 years of initial adjuvant tamoxifen therapy.

Exemestane has shown benefit in terms of disease-free survival when given as an alternative to tamoxifen after initial adjuvant treatment with tamoxifen for 2 - 3 years. It offers an alternative to

tamoxifen after initial adjuvant treatment with tamoxifen for 2 - 3 years and has a different adverse effects profile. Treatment with exemestane is restricted to initiation by a breast ca n ce r

specialist.

Added to the LJF as a prescribing note.

August 2010



09 September 2020


39/97

Product

Manufacturer





Date of NHS Lothian

decision

febuxostat 120mg film-coated tablet

(Adenuric®)

A. MenariniFarmaceuticaInternazionale

SRL

13.06.16 SMC Report No. 1153/16

febuxostat film-coated tablet (Adenuric®) is accepted for restricted use within NHS Scotla n d fo r

the prevention and treatment of hyperuricaemia in adult patients undergoing chemothera p y fo r haematologic malignancies at intermediate to high risk of Tumour Lysis Syndrome (TLS).

SMC restriction: prevention of hyperuricaemia in adult patients at intermediate risk of TLS in

whom allopurinol is either unsuitable or contraindicated, such as:

• Those intolerant of allopurinol

• Those in whom allopurinol is contraindicated, e.g. patients with renal impairment

In a phase III, randomised, double-blind study in adults with haematologic malignancies at

intermediate to high risk of TLS, febuxostat was significantly superior to a xanthine oxidase inhibitor at reducing serum uric acid levels.

Not included on the LJF, because

clinicians have not responded to an invitation to apply for formulary inclusion

for this medicine.

July 2016

fludarabine, 10mg tablet and 50mg for

injection or infusion (Fludara®)

Schering Health Care Ltd

13.11.06


fludarabine phosphate (Fludara®) is accepted for restricted use within NHS Scotland for the

treatment of B-cell chronic lymphocytic leukaemia (CLL) in patients with sufficient bone ma rro w reserves. First line treatment should only be initiated in patients with advanced disease, Rai

stages III/IV (Binet stage C), or Rai stages I/II (Binet stage A/B) where the patient has d ise a se related symptoms or evidence of progressive disease.

Fludarabine phosphate has been associated with higher response rates than ch lo ra mb u cil in clinical trials. No overall survival advantage over other therapies has been demonstrated.

Fludarabine is restricted to use by specialists in haemato-oncology.


Use only.

July 2009

FOLFIRINOX combination chemotherapy (Folic acid, 5-Fluorouracil, Irinotecan,

Oxaliplatin) Various


For use in palliative chemotherapy for inoperable pancreatic adenocarcinoma in good performance status patients.


December 2011

5-FU, irinotectan, oxaliplatin (modified

Folfirinox regimen)


As adjuvant treatment following potentially curative resection of pancreatic cancer. Routinely available in line with local

guidance, Included on the Additiona l L ist for Specialist Use only.

CategorisedRED under the ADTC “Policy

for the use of unlicensed (and off-label use) Medicines in NHS Lothian. Included

on the Additional List for Specialist Use only.

July 2019

fosaprepitant, 115mg powder for solutio n for infusion (Ivemend

®)

Merck Sharp & Dohme Limited

13.10.08 SMC Report No. 506/08

fosaprepitant (Ivemend®) is accepted for restricted use within NHS Scotland for the prevention of

acute and delayed nausea and vomiting associated with highly emetogenic cisplatin -based

cancer chemotherapy. Fosaprepitant is marginally more expensive than aprepitant. It is restricted to use in patients fo r

whom aprepitant is indicated but the oral formulation is not appropriate. Prescribing sh o u ld b e initiated by hospital based specialists only.

Fosaprepitant is also licensed for the prevention of nausea and vomiting associated with moderately emetogenic cancer chemotherapy. As the manufacturer’s submission related only to

its use with highly emetogenic cancer chemotherapy, SMC cannot recommend its use in this setting.


July 2009

Withdrawn from the UK market, February 2011



09 September 2020


40/97

Product

Manufacturer





Date of NHS Lothian

decision

fosaprepitantdimeglumine 150 mg

powder for solution for infusion (IVEmend

®)

MSD Ltd

07.03.11 SMC Report No. 678/11

PRODUCT UPDATE ABBREVIATED SUBMISSION

fosaprepitantdimeglumine (IVEmend 150mg®) is accepted for use within NHS Scotland.

Indication under review: Prevention of acute and delayed nausea and vomiting associate d with highly emetogenic cisplatin based cancer chemotherapy in adults.

IVEmend 150 mg is given as part of a combination therapy. Fosaprepitant 150mg is not recommended for the prevention of nausea and vomiting associated

with moderately emetogenic cancer chemotherapy in adults, as SMC has previously not recommended both fosaprepitant iv and aprepitant capsules for this indication.


Use only.

March 2011

fosaprepitant 150mg powder for solu t io n

for infusion (Ivemend®)

Merck Sharp & Dohme

12.11.18



fosaprepitant (Ivemend 150mg®) is accepted for use within NHSScotland.

Indication under review: prevention of nausea and vomiting associated with highly and moderately emetogenic cancer chemotherapy in paediatric patients aged 6 months to 17 years.

Fosaprepitant is given as part of a combination therapy. SMC has previously accepted fosaprepitant as part of combination therapy for the prevention o f

acute and delayed nausea and vomiting associated with highly emetogenic cisplatin -based chemotherapy in adults (678/11).

SMC has previously accepted aprepitant for use as part of combination therapy for the prevention of nausea and vomiting associated with moderately and highly emetog en ic ca n ce r

chemotherapy in children, toddlers and infants from the age of six months to 17 years (1252 /1 7 and 1241/17 respectively). Intravenous fosaprepitant is a pro-drug of oral aprepitant and it offers

an alternative with limited budget impact.



November 2018

fulvestrant 250mg solution for injection (Faslodex

®)


11.12.17 SMC Report No. 1294/17

NON SUBMISSION

NOT RECOMMENDED: fulvestrant (Faslodex®) is not recommended for use within NHS

Scotland.

Indication under review: Treatment of oestrogen receptor positive, locally advanced or metastatic breast cancer in postmenopausal women not previously treated with endocrine

therapy. The holder of the marketing authorisation has not made a submission to SMC regarding this

product in this setting. As a result we cannot recommend its use within NHS Scotland.

NOT RECOMMENDED

fulvestrant 250mg solution for injection (Faslodex

®)


08.02.16 SMC Report No. 114/04


fulvestrant (Faslodex®) is accepted for use within NHS Scotland.

Indication under review: for the treatment of postmenopausal women with oestro g en re ce p to r

positive, locally advanced or metastatic breast cancer for disease relapse on or afte r a d ju va nt anti-oestrogen therapy, or disease progression on therapy with an anti-oestrogen.

In a phase III randomised double blind study, fulvestrant 500mg increased progression free survival and overall survival compared to fulvestrant 250mg.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of fulvestrant. This advice is contingent upon the continuing availability o f


meeting

Included on the Additional List, Specia list Use only, for the indication in question.

October 2016



09 September 2020


41/97

Product

Manufacturer





Date of NHS Lothian

decision

gefitinib 250mg film-coated tablets

(Iressa®)

AstraZeneca UK Ltd

07.12.15

SMC Report No: 615/10 2

nd RESUBMISSION

Patient AccessScheme

gefitinib (Iressa®) is accepted for restricted use within NHS Scotland.

Indication under review: the treatment of adult patients with locally advanced or metastatic n o n-small cell lung cancer (NSCLC) with activating mutations of epidermal growth factor receptor

tyrosine kinase (EGFR-TK). SMC restriction: in patients with previously untreated locally advanced or metastatic NSCLC with

activating EGFR-TK mutations i.e. as a first-line therapy. In patients with EGFR mutation-positive, advanced NSCLC, randomised controlled studies

demonstrated an improvement in the progression-free survival and tumour respon se ra te s fo r those treated with gefitinib compared with platinum-doublet chemotherapy. There was no overall

survival benefit demonstrated. This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e

cost-effectiveness of gefitinib. This advice is contingent upon the continuing availabil it y o f th e patient access scheme in NHS Scotland or a list price that is equivalent or lower.

Not included on the LJF, because

clinicians have not responded to an invitation to apply for formulary inclusion

for this medicine.

March 2016

gemcitabine (Gemzar®)

Eli Lilly & Co Ltd


Biliary tract cancer.




September 2005

gemcitabine 200mg and 1g powder for

solution for infusion (Gemzar®)

Eli Lilly and Company Ltd

11.12.06


gemcitabine (Gemzar®), in combination with paclitaxel, is accepted for restricted use within NHS

Scotland for the treatment of patients with metastatic breast cancer who have relapsed following adjuvant/neoadjuvant chemotherapy. Prior chemotherapy should have included an

anthracycline unless clinically contraindicated. Gemcitabine in combination with paclitaxel modestly improves outcomes, compared to paclitaxel

monotherapy, in those previously treated with an anthracycline. For this indication gemcitabine is restricted to use by oncologists specialising in the treatment of

breast cancer.


Use only.

December 2007

gemcitabine (Gemzar®)


In combination with dexamethasone and cisplatin for relapsed / refractory lymphoma.


Use only.



May 2014

gemcitabine in combination with carboplatin

April 2016

NICE MTA 389

NOT RECOMMENDED: NICE 389 Topotecan, pegylated liposomal doxorubicin hydrochlorid e , paclitaxel, trabectedin and gemcitabine for treating recurrent ovarian cancer

Gemcitabine in combination with carboplatin is not recommended within its marketing authorisation for treating the first recurrence of platinum-sensitive ovarian cancer. The Appraisal

Committee was unable to make recommendations on the use of these technologies for treat in g platinum-sensitive ovarian cancer beyond the first recurrence.

NOT RECOMMENDED

gemcitabine powder for infusion and

capecitabine tablets


Adjuvant treatment following potentially curative resection of pancreatic cancer. Routinely available in line with local or




July 2017



09 September 2020


42/97

Product

Manufacturer





Date of NHS Lothian

decision

gemtuzumabozogamicin 5mg powder fo r

concentrate for solution for infusion (Mylotarg

®)

Pfizer Ltd

08.10.18 SMC Report No. 2089

Following a full submission assessed under the end of life and orphan medicine process

gemtuzumab ozogamicin (Mylotarg®) is accepted for restricted use within NHSScotland for

combination therapy with daunorubicin and cytarabine for the treatment of patients age 15 years

and above with previously untreated, de novo CD33-positive acute myeloid leuka e mia (AML ), except acute promyelocytic leukaemia (APL).

SMC restriction: use in patients with a favourable, intermediate or unknown cytogenetic profile. In an open-label, phase III study of adults with AML, the addition of gemtuzumab ozogamicin to

standard intensive chemotherapy was associated with significant improvement in event -free survival compared with standard intensive chemotherapy alone. Events included failure to

achieve remission with induction therapy, relapse of disease, or death. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.



December 2018

gilteritinib 40mg film-coated tablets

(Xospata®)

Astellas Pharma Ltd

07.09.20


Following a full submission assessed under the end of life and orphan medicine process

gilteritinib (Xospata®) is accepted for use within NHSScotland.

Indication under review: as monotherapy for the treatment of adult patients who have relapsed

or refractory acute myeloid leukaemia with a FLT3 mutation. In an open-label, phase III study, gilteritinib improved overall survival compared with salvage

chemotherapy in patients with relapsed or refractory acute myeloid leukaemia with a FLT3 mutation.

This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based,

or a PAS/ list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.

Formulary classification not yet decided –

waiting for information from clinician.

granisetron 3.1mg / 24 hours transdermal

patch (Sancuso®)

ProStrakan Ltd

07.10.13



granisetron 3.1mg / 24 hours transdermal patch (Sancuso®) is accepted for use within NHS

Scotland in adults for the prevention of nausea and vomiting associated with moderately or highly emetogenic chemotherapy, for a planned duration of 3 to 5 consecutive days, where o ra l

anti-emetic administration is complicated by factors making swallowing difficult. Granisetron 3.1mg / 24 hours transdermal patch is slightly more expensive than the oral

formulation. It provides an alternative option in patients who have difficulty swallowing oral medication.



October 2013

histamine dihydrochloride, 500

microgram/0.5mL, vial (Ceplene®)

Meda Pharmaceuticals Ltd

17.01.11


NOT RECOMMENDED: histamine dihydrochloride (Ceplene®) is not recommended for use

within NHS Scotland. Indication under review: maintenance therapy for adult patients with acute myeloid leukaemia in

first remission concomitantly treated with interleukin-2. The efficacy of histamine dihydrochloride has not been fully demonstrated in patients older than age 60 years.

In a randomised open-label study, histamine plus interleukin-2 was superior to no treatment fo r the endpoint of leukaemia free survival (LFS) in a sub-group of patients in first complete

remission. In post hoc analysis of patients in first complete remission and aged less than 60 years, LFS rates at 36 months were 50% versus 30%.

Overall the manufacturer did not present a sufficiently robust clinical or economic ca se to g a in acceptance by SMC.

NOT RECOMMENDED

histidine - tryptophan - ketoglutarate

(HTK) (Custodial®)

Kohler Medical Ltd


(1) Non heart-beating kidney - liver donors (NHBD)

(2) Live donor liver transplantation (LDLT)


Use only.

May 2006



09 September 2020


43/97

Product

Manufacturer





Date of NHS Lothian

decision

histrelin acetate, 50mg subcutaneous

implant (Vantas®)

Orion Pharma (UK) Ltd

10.08.09


histrelin (Vantas®) subcutaneous implant is accepted for restricted use within NHS Scotlan d fo r

palliative treatment of advanced prostate cancer. Histrelin is restricted to use in patients with an anticipated life expectancy of at least one year in

whom annual administration will offer advantages. In a single-arm study, histrelin provided effective suppression of testosterone levels in p a t ie n ts

with advanced prostate cancer. It requires less frequent administration than othe r lu te in is i n g hormone releasing hormone (LHRH) agonists. Other LHRH agonists are available at a lower

acquisition cost.

‘Not preferred’ in Lothian as suitable

alternatives exist.

January 2010

hydroxycarbamide (Siklos®)

Nordic Pharma UK

12.10.09 SMC Report No. 582/09

NON SUBMISSION

NOT RECOMMENDED: hydroxycarbamide (Siklos®) is not recommended for use within NHS

Scotland for the prevention of recurrent painful vaso-occlusive crises including acute chest

syndrome in paediatric and adult patients suffering from symptomatic Sickle Cell Syndrome. The holder of the marketing authorisation has not made a submission to SMC regarding this

product in this indication. As a result we cannot recommend its use within NHS Scotland

NOT RECOMMENDED

hydroxycarbamide (Xromi®)

100mg/mLoral solution

Nova Laboratories Limited



hydroxycarbamide oral solution (Xromi®) is accepted for restricted use within NHSScotland.

Indication under review: for the prevention of vaso-occlusive complications of Sickle Cell

Disease in patients over 2 years of age. SMC restriction: children who are too young to be able to swallow capsules / tablets and adults

and adolescents who have difficulty in swallowing solid oral dosage forms. The availability of hydroxycarbamide oral solution (Xromi

®) provides a licensed alternative to

unlicensed liquid preparations.

Routinely available in line with national guidance. Included on the Additional List ,

for Specialist Use only. All prescribing an d supply is via the specialist paediatric

haematology service.

August 2020

ibandronic acid (Bondronat®)

Roche

11.10.04


ibandronic acid is accepted for use within NHS Scotland for the prevention of ske le tal e ve n ts (pathological fractures, bone complications requiring radiotherapy or surgery) in patients with

breast cancer and bone metastases. It reduces the rate of skeletal events consisting of a composite of vertebral fractures,

pathological non-vertebral fractures and the need for radiotherapy or surgery to deal with b o n e complications. It can be given both by the oral or intravenous route.

Added to the Formulary as first choice bisphosphonate, on the advice of an

oncologist/ haematologist, for the prevention of skeletal related events in

patients with breast cancer.

February 2005

ibritumomab tiuxetan (Zevalin®)

Schering Health Care Ltd

09.07.07 SMC Report No. 171/05

RESUBMISSION

NOT RECOMMENDED: ibritumomab tiuxetan (Zevalin®) is not recommended for use within

NHS Scotland for the preparation of a radiopharmaceutical incorporating Yttrium 90 [90

Y] for th e treatment of adult patients with rituximab relapsed or refractory CD20+ follicular B-cell non-

Hodgkin's lymphoma (NHL). The manufacturer did not present a sufficiently robust economic analysis to gain acceptance b y

SMC.

NOT RECOMMENDED

ibritumomab tiuxetan 1.6mg/mL (Zevalin

®)

Bayer plc

11.08.08 SMC Report No. 449/08

NON SUBMISSION

NOT RECOMMENDED: ibritumomab tiuxetan (Zevalin) is not recommended for use within NHS Scotland as consolidation therapy after remission induction in previously untreated patients with

follicular lymphoma. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED



09 September 2020


44/97

Product

Manufacturer





Date of NHS Lothian

decision

ibrutinib 140mg hard capsule (Imbruvica®)

Janssen-Cilag Ltd.

08.08.16 SMC Report No. 1150/16


ibrutinib (Imbruvica®) is accepted for use within NHS Scotland for the treatment of adult patients

with relapsed or refractory mantle cell lymphoma (MCL). In a randomised, open-label, phase III study ibrutinib significantly prolonged progression-free

survival, the primary endpoint, compared to a chemotherapy treatment, in patients with relapsed or refractory MCL.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of ibrutinib. This advice is contingent upon the continuing availability of

the PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.



November 2016

ibrutinib 140mg hard capsules

(Imbruvica®)

Janssen-Cilag Ltd.

10.04.17



Following a resubmission assessed under the end of life and orphan medicine process:

ibrutinib (Imbruvica®) is accepted for restricted use within NHS Scotland.

Indication under review: the treatment of adult patients with chronic lymphocytic leukaemia

(CLL) who have received at least one prior therapy. SMC restriction: patients with relapsed/refractory CLL and for whom fludarabine-based regimens

are inappropriate. In an open-label, phase III study, ibrutinib significantly increased progression-free survival

compared with an anti-CD20 antibody in patients with relapsed or refractory CLL. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of ibrutinib. This advice is contingent upon the continuing ava ila bil it y o f the PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account o f

views from a Patient and Clinician Engagement (PACE) meeting. This resubmission relates to use as a single agent for the treatment of adult patien ts with CL L

who have received at least one prior therapy. SMC published advice in August 2016 that ibrutinib was accepted for restricted use as a single agent for patients with 17p deletion or TP53

mutation who are unsuitable for chemo-immunotherapy (SMC 1151/16).



July 2017

ibrutinib (Imbruvica®) 140mg hard

capsules

Janssen-Cilag Ltd

12.06.17 SMC Report No. 1258/17

NON SUBMISSION

NOT RECOMMENDED: ibrutinib (Imbruvica®) is not recommended for use within NHS Scotland.

Indication under review: In combination with bendamustine and rituximab for the t re a tme n t o f

adult patients with chronic lymphocytic leukaemia who have received at least one prior therapy. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

ibrutinib 140mg hard capsules

(Imbruvica®)

Janssen-Cilag Ltd

13.11.17


NOT RECOMMENDED: ibrutinib (Imbruvica®) is not recommended for use within NHS Scotland.

Indication under review: As a single agent for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (who do not have 17p deletion or TP53 mutation).

The holder of the marketing authorisation has not made a submission to SMC regarding this product in this setting. As a result we cannot recommend its use within NHS Scotland.

NOT RECOMMENDED

ibrutinib 140mg hard capsules and

140mg, 280mg, 420mg and 560mg film-coated tablets (Imbruvica

®)

Janssen-Cilag Ltd

11.11.19 SMC Report No. 2244

NON SUBMISSION

NOT RECOMMENDED: ibrutinib (Imbruvica®) is not recommended for use within NHSScotland.

Indication under review: In combination with obinutuzumab for the treatment of a d ult p a t ie nts with previously untreated chronic lymphocytic leukaemia.


NOT RECOMMENDED



09 September 2020


45/97

Product

Manufacturer





Date of NHS Lothian

decision

ibrutinib 140mg hard capsules and

140mg, 280mg, 420mg and 560mg film-coated tablets (Imbruvica

®)

Janssen-Cilag Ltd

11.11.19 SMC Report No. 2245

NON SUBMISSION

NOT RECOMMENDED: ibrutinib (Imbruvica®) is not recommended for use within NHSScotland.

Indication under review: As a single agent for the treatment of adult patients with Waldenström’smacroglobulinaemia who have received at least one prior therapy, or in first l in e

treatment for patients unsuitable for chemo-immunotherapy. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

idelalisib 100mg and 150mg tablets (Zydelig

®)

Gilead Sciences Ltd.

09.03.15 SMC Report No. 1026/15


idelalisib (Zydelig®) is accepted for restricted for use within NHS Scotland in combination with

rituximab for the treatment of adult patients with chronic lymphocytic leukaemia (CLL):

• who have received at least one prior therapy, or • as first line treatment in the presence of 17p deletion or TP53 mutation in patients

unsuitable for chemo-immunotherapy. SMC restriction: patients with relapsed CLL who are unsuitable for chemotherapy and treatment

naïve patients with 17p deletion or TP53 mutation who are unsuitable for chemo -immunotherapy.

Idelalisib in combination with an anti-CD20 antibody significantly improves progression free survival compared with an anti-CD20 antibody alone in patients with relapsed CLL. The

treatment effect across subgroups with 17p deletion and/or TP53 mutation was consiste n t with that of the total study population.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of idelalisib. It is contingent upon the continuing availability of the patie n t

access scheme in NHS Scotland or a list price that is equivalent or lower.

Included on the Additional List, Specia list Use only.

September 2015

idelalisib, 100mg and 150mg film-coated tablets (Zydelig

®)

Gilead Sciences Ltd.

11.05.15 SMC Report No. 1039/15


idelalisib (Zydelig®) is accepted for use within NHS Scotland.

Indication under review: Monotherapy for the treatment of adult patients with follicular lymphoma

(FL) that is refractory to two prior lines of treatment. Idelalisib demonstrated clinical activity, measured by overall response rate, in a p h a se I I n o n -

comparative study. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of idelalisib and is contingent upon the continuing availability of th e PAS in NHS Scotland or a list price that is equivalent or lower.


Includedon the Additional List, Specialist Use only, for the indication in question.

December 2015

idelalisib (Zydelig®) 100mg, 150mg film-

coated tablets

Gilead Sciences Ltd

12.12.16 SMC Report No. 1212/16

NON SUBMISSION

NOT RECOMMENDED: idelalisib (Zydelig®) is not recommended for use within NHS Scotland in

combination with ofatumumab for the treatment of adult patients with chronic lymphocytic

leukaemia: • who have received at least one prior therapy, or

• first line treatment in the presence of 17p deletion or TP53 mutation in patients who are no t eligible for any other therapies.

The holder of the marketing authorisation has not made a submission to SMC regardin g this product in this indication. As a result we cannot recommend its use within NHS Scotland.

NOT RECOMMENDED

imatinib (Glivec) Novartis

April 2012

NICE MTA 251 supersedes SMC Report No. 01/02 and SMC Report No. 26/02

NICE MTA 251 advised that standard-dose imatinib (400mg per day for patients in chronic

phase) is recommended as an option for the first-line treatment of adults with chronic phase Philadelphia-chromosome-positive chronic myeloid leukaemia (CML).

The Scottish Medicines Consortium (SMC) has previously accepted imatinib and nilotinib for the treatment of adult patients with newly diagnosed Philadelphia-chromosome-positive CML in th e

chronic phase. NICE MTA 251 was withdrawn in December 2016 but the NICE MTA 251 advice for imatinib

remains valid in NHSScotland.

Approved for use - added to the Additional

List.

January 2003



09 September 2020


46/97

Product

Manufacturer





Date of NHS Lothian

decision

imatinib (Glivec) Novartis

09.08.03


imatinib (Glivec) is recommended for restricted use within the NHS in Scotland, under the supervision of an oncologist for patients with Kit-positive gastrointestinal stromal tumours

(GIST).

Approved for use - added to the Additional

List.

imatinib (Glivec®)


10.12.07 SMC Report No. 426/07

NON SUBMISSION

NOT RECOMMENDED:imatinib (Glivec®) is not recommended for use within NHS Scotla n d fo r

the treatment of adult patients with relapsed or refractory Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) as monotherapy.


NOT RECOMMENDED

imatinib (Glivec®)


10.12.07



the treatment of adult patients with newly diagnosed Philadelphia chromosome posit ive a cu te

lymphoblastic leukaemia (PH + ALL) in combination with chemotherapy. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

imatinib (Glivec®)


10.12.07 SMC Report No. 428/07

NON SUBMISSION


the treatment of adult patients with myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with platelet-derived growth factor receptor (PDGFR) gene re-arrangements.


NOT RECOMMENDED

imatinib (Glivec®)


10.12.07



the treatment of adult patients with advanced hypereosinophilic syndrome (HES) and/or chron ic

eosinophilic leukaemia (CEL) with FIP1L1-PDGFRα rearrangement. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

imatinib (Glivec®)


10.12.07



the treatment of adult patients with unresectable dermatofibrosarcoma protuberans (DFSP) a n d

adult patients with recurrent and/or metastatic DFSP who are not e ligible for surgery. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

imatinib 100mg and 400mg film-coated

tablets (Glivec®)


07.12.09


imatinib (Glivec®) is accepted for restricted use within NHS Scotland.

Indication under review: adjuvant treatment of adult patients who are at significant risk of relapse following resection of Kit (CD117)-positive gastrointestinal stromal tumours (GIST). Patients who

have a low or very low risk of recurrence should not receive adjuvant treatment. SMC restriction: Imatinib is restricted to use in patients at high risk of recurrence following

complete resection (according to the Armed Forces Institute of Pathology (AFIP) risk criteria). Imatinib, given for a period of one year, significantly improved the estimated one year

recurrence-free survival compared with placebo and was associated with an increase of 16.4 months in median time to recurrence in patients at high risk of relapse following resection.

The economic case was demonstrated for a one-year adjuvant treatment duration only.


Use only.

April 2011



09 September 2020


47/97

Product

Manufacturer





Date of NHS Lothian

decision


tablets (Glivec®)


09.04.12


nd RESUBMISSION

imatinib (Glivec®) is accepted for restricted use within NHS Scotland.

Indication under review: adjuvant treatment of adult patients who are at significant risk of relapse following resection of Kit (CD117)-positive gastrointestinal stromal tumours (GIST). Patients who

have a low or very low risk of recurrence should not receive adjuvant treatment. SMC restriction: Imatinib is restricted to use in patients at high risk of recurrence following

complete resection (according to the Armed Forces Institute of Pathology (AFIP) risk criteria). Adjuvant imatinib therapy given for a period of three years compared to one year, sig n if ica n tly

improved the recurrence free survival in adult patients at significant risk of relapse following resection of GIST.

The clinical and cost-effectiveness of three years adjuvant imatinib treatment was demonstrated.

Includedon the Additional List, Specialist


See SMC advice above. Same SMC Report Number – this advice relates to a n

extension in treatment length to 3 years.

July 2012

imatinib 100mg and 400mg film-coated tablets (Glivec

®)


January 2012 NICE MTA 241

NOT RECOMMENDED: NICE MTA 241 Dasatinib, high-dose imatinib and nilotinib for the treatment of imatinib-resistant chronic myeloid leukaemia (CML) (part review of NICE technology

appraisal guidance 70), and dasatinib and nilotinib for people with CML for whom treatment with imatinib has failed because of intolerance states that high-dose imatinib is not recommended for

the treatment of chronic, accelerated or blast-crisis phase Philadelphia-chromosome-positive CML that is resistant to standard-dose imatinib.

NOT RECOMMENDED


tablets(Glivec®)


07.10.13


NOT RECOMMENDED: matinib (Glivec®) is not recommended for use within NHS Scotlan d fo r

the treatment of paediatric patients with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) integrated with chemotherapy.


NOT RECOMMENDED

imiquimod 5% cream (Aldara®)

3M Health Care

09.05.05


imiquimod 5% (Aldara) is accepted for restricted use within NHS Scotland for the topical treatment of small superficial Basal Cell Carcinoma in adult patients in whom standard treatment with surgery or cryotherapy is contraindicated.

Its use should be supervised by specialists in dermatology. At 12 weeks post treatment the composite clearance rates in the randomised con tro lle d t ria ls

were between 73-77% and initial clearance rates in the open label studies we re b e twe e n 9 0 -94%. There is only limited follow-up data beyond 12 months.

Added to the LJF as a prescribing note.

September 2005

inotuzumabozogamicin 1mg powder for

concentrate for solution for infusion (BESPONSA

®)

04.05.18


Following a full submission assessed under the ultra-orphan and end of life medicine process, i

inotuzumabozogamicin (BESPONSA®) is accepted for restricted use within NHSScotland.

Indication under review: as monotherapy for the treatment of adults with relapsed or refractory

CD22-positive B cell precursor acute lymphoblastic leukaemia (ALL). Adult patients with Philadelphia chromosome positive relapsed or refractory B cell precursor ALL should have failed

treatment with at least one tyrosine kinase inhibitor. SMC restriction: in patients for whom the intent is to proceed to stem cell transplantation.

A phase III open label randomised controlled study demonstrated improvements in re missio n rates and overall survival for the patient population under review when treated with

inotuzumabozogamicin compared with standard chemotherapy. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of inotuzumabozogamicin. This advice is contingent upon the contin u ing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.



October 2018



09 September 2020


48/97

Product

Manufacturer





Date of NHS Lothian

decision

ipilimumab 5mg/mL concentrate for

solution for infusion (Yervoy®)

Bristol-Myers Squibb

08.04.13



ipilimumab (Yervoy®) is accepted for use within NHS Scotland for the treatment of advanced

(unresectable or metastatic) melanoma in adults who have received prior therapy. Ipilimumab demonstrated a survival benefit over an investigational glycoprotein100 peptide

vaccine in previously treated patients with advanced melanoma. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of ipilumumab. This SMC advice is contingent upon the continuing availability of the patient access scheme or a list price that is equivalent or lower.



June 2013

ipilimumab 5mg/mL concentrate for solution for infusion (Yervoy

®)


10.11.14 SMC Report No. 997/14


ipilimumab (Yervoy®) is accepted for use within NHS Scotland for the treatment of advanced

(unresectable or metastatic) melanoma in adults (first-line use).

In a phase III, randomised study median overall survival was extended by 2.1 months in patients treated with ipilimumab plus dacarbazine (an unlicensed dose regimen) compared with

dacarbazine alone. Efficacy data for the licensed dose of ipilimumab are limited to two retrospective single-arm observational studies where median overall survival was 11.5 to 1 4 .3

months. This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e

cost-effectiveness of Ipilimumab. This advice is contingent upon the continuing availability of the Patient Access Scheme in NHS Scotland or a list price that is equivalent or lower.



question.

December 2014

ipilimumab 5mg/mL concentrate for

solution for infusion (Yervoy®)

Bristol-Myers Squibb Pharmaceuticals UK

08.10.18



ipilimumab (Yervoy®) is accepted for use within NHSScotlandasmonotherapyfor the treatment of

advanced (unresectable or metastatic) melanoma in adolescents 12 years of age and older. SMC has previously accepted ipilimumab for the treatment of advanced (unresectable or

metastatic) melanoma in previously untreated adult patients (SMC 997/14) and in a d u lts wh o have received prior therapy (SMC 779/12).

Ipilimumab was accepted for use in previously untreated patients (SMC 997/14) following a submission under the end of life and orphan process.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of ipilimumab. This SMC advice is contingent upon the continuing

availability of the patient access scheme or a list price that is equivalent or lower.



November 2018

ixazomib 2.3mg, 3mg and 4mg hard

capsules(Ninlaro®)

Takeda UK Ltd

09.07.18


NOT RECOMMENDED: ixazomib (Ninlaro®) is not recommended for use within NHS Scotland.

Indication under review:in combination with lenalidomide and dexamethasone for the treatme nt of adult patients with multiple myeloma who have received at least one prior therapy.


NOT RECOMMENDED

lanadelumab 300mg solution for injection

(Takhzyro®)

Shire, part of Takeda UK Ltd

09.12.19


lanadelumab (Takhzyro®) is accepted for restricted use within NHSScotland.

Indication under review: For the routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older.

SMC restriction: patients with HAE type I or II, who would otherwise be considered for long-term prophylaxis treatment with C1-esterase inhibitor.

In a phase III study in patients with HAE, lanadelumab reduced the rate of angioedema atta cks compared with placebo.


or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.




January 2020



09 September 2020


49/97

Product

Manufacturer





Date of NHS Lothian

decision

lanreotide (Somatuline® LA)

Ipsen Ltd

11.12.06 SMC Report No. 231/06

NON SUBMISSION

NOT RECOMMENDED:lanreotide (Somatuline® LA) is not recommended for use within NHS

Scotland for the treatment of thyrotrophic adenomas when the circulating level of thyroid stimulating hormone remains inappropriately high after surgery and/or radiotherapy.


NOT RECOMMENDED

lanreotide(Somatuline®Autogel)


Management of neuroendocrine tumours. Included on the LJF, as first choice, for the indication in question.


guidance. Included in the LJF, Specialist Initiation only.

March 2016

As per LJF

March 2019

lapatinib, 250mg film-coated tablets

(Tyverb®)

GlaxoSmithKline

13.07.10


NOT RECOMMENDED:lapatinib (Tyverb®) is not recommended for use within NHS Scotland.

Indication under review: in combination with capecitabine, for the treatment of patients with advanced or metastatic breast cancer whose tumours overexpress ErbB2 (HER2) and who have

progressive disease following prior therapy including anthracyclines and taxanes a n d th e ra py with trastuzumab in the metastatic setting.

In a randomised open-label study the median time to progression for lapatinib plus capecitabin e was significantly longer than for capecitabine monotherapy. There was no statistically significant

difference in overall survival. Compared with capecitabine monotherapy, the manufacturer’s justification of the treatment’s

cost in relation to its health benefits was not sufficient to gain acceptance by SMC. Th e re wa s also uncertainty about the comparative effectiveness and cost-effectiveness compared to

unlicensed use of trastuzumab and capecitabine in patients with metastatic disease confined to the central nervous system, itself a treatment of unproven cost-effectiveness.

NOT RECOMMENDED

lapatinib (Tyverb®) 250 mg film-coated

tablets

GlaxoSmithKline

June 2012 NICE MTA 257 supersedes SMC Report

No. 768/12

NOT RECOMMENDED: NICE MTA 257 states that lapatinib in combination with an aroma ta se inhibitor is not recommended for first-line treatment in postmenopausal women with meta sta t ic

hormone-receptor-positive breast cancer that overexpresses human epidermal growth factor receptor 2 (HER2).

NOT RECOMMENDED

lapatinib (Tyverb®) 250 mg film-coated

tablets

GlaxoSmithKline

11.11.13 SMC Report No. 925/13

NON SUBMISSION

NOT RECOMMENDED: lapatinib (Tyverb®) is not recommended for use within NHS Scotland

for the treatment of adult patients with breast cancer, whose tumours overexpress HER2

(ErbB2) in combination with trastuzumab for patients with hormone receptor-negative metastatic disease that has progressed on prior trastuzumab therapy(ies) in combination with

chemotherapy. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED



09 September 2020


50/97

Product

Manufacturer





Date of NHS Lothian

decision

lenalidomide, 7.5mg, 10mg, 15mg and

25mg hard capsules (Revlimid®)

Celgene Limited

07.04.14


nd RESUBMISSION

lenalidomide (Revlimid®) is accepted for restricted use within NHS Scotland in combination with

dexamethasone, for the treatment of multiple myeloma in adult patients who have re ce ive d a t least one prior therapy. (This resubmission relates to patients who have received only one p rio r

therapy). SMC restriction: to use at first relapse in patients who have received prior therapy with

bortezomib in whom thalidomide has not been tolerated or is contraindicated. Lenalidomide plus dexamethasone significantly increased the time to progression compared

with dexamethasone alone in multiple myeloma patients who had been treated with at least on e prior therapy.

SMC has previously accepted lenalidomide for use in patients who have received a t le a st two prior lines of therapy i.e. at second relapse. This advice now extends its use to patients a t f irst

relapse who received bortezomib as their one prior therapy.

For use in patients who have received at

least two prior lines of therapy. Added to the Additional List, for Specialist Use only.

For patients who have received only one

prior therapy. Not included on the LJF, pending protocol.

September 2010

July 2014

lenalidomide, 2.5mg, 5mg, 7.5mg, 10mg, 15mg, 20mg and 25mg

capsules(Revlimid®)

Celgene Europe Limited

07.12.15


lenalidomide (Revlimid®) is accepted for restricted use within NHS Scotland.

Indication under review: treatment of adult patients with previously untreated multiple mye lo ma

who are not eligible for transplant. SMC restriction: for use in patients unsuitable for thalidomide-containing regimens.

Continuous lenalidomide plus low-dose dexamethasone, compared with melphalan, prednisolone plus thalidomide, significantly improved progression-free survival in treatment-

naive patients with newly diagnosed multiple myeloma who were not eligible for transplant. Overall survival data are immature, but interim analyses suggest a survival benefit for

lenalidomide plus low-dose dexamethasone compared with melphalan, prednisolone plus thalidomide.

This submission focuses on lenalidomide in combination with dexamethasone. Lenalidomid e is also licensed for use in combination with melphalan and prednisolone for the treatment of a d ult

patients with previously untreated multiple myeloma who are not eligible for transplant. The submitting company did not provide evidence for SMC assessment therefore SMC cannot

recommend this combination for use in this treatment setting. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.

Included on the Additional List, Specialist Use only, for the indication in question.

April 2016

lenalidomide (Revlimid®) 2.5mg, 5mg,

7.5mg, 10mg, 15mg, 20mg and 25mg

hard capsules Celgene Ltd

07.11.16


NOT RECOMMENDED: lenalidomide (Revlimid®) is not recommended for use within NHS

Scotland for the treatment of adult patients with relapsed or refractory mantle cell lymphoma.


NOT RECOMMENDED

lenalidomide 2.5mg, 5mg, 7.5mg, 10mg, 15mg, 20mg and 25mg hard capsules

(Revlimid®)

Celgene Ltd

08.10.18

SMC Report No.2125 NON SUBMISSION

NOT RECOMMENDED:lenalidomide (Revlimid®) is not recommended for use within

NHSScotland as monotherapy for the maintenance treatment of adult patients with newly

diagnosed multiple myeloma who have undergone autologous stem cell transplantation. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED



09 September 2020


51/97

Product

Manufacturer





Date of NHS Lothian

decision

lenalidomide 2.5mg, 5mg, 7.5mg, 10mg,

15mg, 20mg and 25mg hard capsules (Revlimid

®)

Celgene Ltd

12.08.19


NOT RECOMMENDED:lenalidomide (Revlimid®) is not recommended for use within

NHSScotland. Indication under review: as combination therapy with bortezomib and dexameth aso ne fo r th e

treatment of adult patients with previously untreated multiple myeloma who are not e lig ib le fo r transplant.

The holder of the marketing authorisation has not made a submission to SMC regarding this product in this indication. As a result we cannot recommend its use within NHSScotland. Note :

SMC advice 1096/15 is still valid.

NOT RECOMMENDED

lenvatinib 4mg and 10mg hard capsules

(Lenvima®)

Eisai Ltd.

10.10.16


lenvatinib (Lenvima®) is accepted for use within NHS Scotland for the treatment of adult patients

with progressive, locally advanced or metastatic, differentiated (papillary/follicular/Hürthle ce ll) thyroid carcinoma (DTC), refractory to radioactive iodine (RAI).

Lenvatinib, compared with placebo, significantly improved progression free survival in adults with RAI-refractory DTC.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of lenvatinib. This advice is contingent upon the continuing availability of




November 2016

lenvatinib 4mg hard capsules

(Lenvima®)

Eisai Ltd

08.04.19


lenvatinib (Lenvima®) is accepted for use within NHSScotland as monotherapy for the treatment

of adult patients with advanced or unresectable hepatocellular carcinoma who have received no prior systemic therapy.

In a phase III study in patients with unresectable hepatocellular carcinoma who had not received treatment for advanced disease, lenvatinib was non-inferior to another multikinase inhib ito r fo r

overall survival. SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves th e

cost effectiveness of lenvatinib and is contingent upon the continuing availability of th e PAS in NHS Scotland or a list price that is equivalent or lower.



July 2019

lenvatinib 4mg and 10mg hard capsules (Kisplyx

®)

Eisai Ltd

11.11.19 SMC Report No. 2199


lenvatinib (Kisplyx®) is accepted for use within NHSScotland.

Indication under review: in combination with everolimus for the treatment of adult patie nts with

advanced renal cell carcinoma (RCC) following one prior vascular endothelial growth factor (VEGF)-targeted therapy

In a phase II study, the addition of lenvatinib to everolimus significantly improved pro g ressio n -free survival in patients with advanced renal cell carcinoma who had received one previous

VEGF-targeted therapy. This SMC advice takes account of the benefit of Patient Access Schemes (PAS) th a t imp ro ve

the cost effectiveness of lenvatinib and everolimus. This advice is contingent upon the continuing availability of these PAS in NHSScotland or list prices that are equivalent or lower.


Routinely available in line with national guidance. Included on the Additional L ist


August 2020



09 September 2020


52/97

Product

Manufacturer





Date of NHS Lothian

decision

letrozole 2.5mg tablets (Femara®)

Novartis Pharmaceuticals (UK) Ltd

07.03.05 SMC Report No. 152/05

letrozole (Femara®) is accepted for restricted use within NHS Scotland for the treatment of

invasive early breast cancer in postmenopausal women who have received prior standard adjuvant tamoxifen therapy.

Treatment should continue for 3 years or until tumour relapse, whichever occurs first. Following 5 years of adjuvant tamoxifen therapy the risk of recurrence (in ipsilateral breast, n e w

tumour in contralateral breast or distance metastases) occurs at an aggregate rate of 2 - 3% p e r year. The use of letrozole as extended adjuvant treatment resulted in a 43% lower risk of

recurrence compared with placebo. However, a significant difference for overall survival, defined as time to death from any cause, was seen in lymph-node positive patients only.

Clinicians and patients should consider the residual risk of recurrence, individual preferences and the risks and benefits of treatment.

Letrozole is restricted to initiation to breast cancer specialists.

Added to the Formulary as a prescribing

note.

August 2005

letrozole 2.5mg tablets (Femara®)


08.05.06


letrozole (Femara) is accepted for restricted use within NHS Scotland for the adjuvant treatment of postmenopausal women with hormone receptor positive invasive early breast

cancer. Letrozole has shown benefit over standard anti-oestrogen therapy in terms of disease-free

survival, although a pre-planned sub-group analysis showed a statistically significant benef ic ia l effect in node-positive but not node-negative patients. It offers an alternative to existing

treatment and has a different range of adverse effects. Another aromatase inhibitor is available for the same indication at a lower cost.

Treatment with letrozole should be initiated by a breast cancer specialist.

Added to the LJF as first choice for patients at risk of early recurrence or with

a contraindication to tamoxifen. Letrozole also first choice for extended adjuvant

treatment after 5 years treatment with tamoxifen.

August 2010

letrozole 2.5mg tablets


For epithelial ovarian cancer. Added to the Additional List, for Specialist initiation.

Categorised AMBER under the ADTC

‘Policy for the use of unlicensed (and off-label use) Medicines in NHS Lothian’.

December 2016

levofloxacin (Tavanic®)

Hoechst Marion Roussel


Antibiotic prophylaxis for neutropenic sepsis in lung cancer patients undergoing chemotherapy

only.

Not preferred as suitable alternatives exist.

December 2006

lipegfilgrastim, 6mg, solution for inject io n (Lonquex

®)

Teva Pharma BV

07.04.14 SMC Report No. 908/13

lipegfilgrastim (Lonquex®) is accepted for restricted use within NHS Scotland for reduction in the

duration of neutropenia and the incidence of febrile neutropenia in adult patien ts t re a te d with

cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).

SMC restriction: where a long-acting granulocyte-colony-stimulating factor is appropriate. In a randomised, double-blind study, in adults with breast cancer given myelosuppressive

chemotherapy associated with a high risk of febrile neutropenia, lipegfilgrastim was co mp a re d with another long-acting granulocyte colony-stimulating factor when used as primary prophylaxis

against febrile neutropenia. The study found lipegfilgrastim was non-inferior to the co mp a ra to r preparation in terms of the mean duration of severe neutropenia in the first chemotherapy cycle.

Included on the Additional List, Specia lis t Use only.

March 2016

liposomal cytarabine 50mg suspension for injection (DepoCyte

®)


13.08.07 SMC Report No. 164/05

RESUBMISSION

NOT RECOMMENDED: liposomal cytarabine suspension (DepoCyte®) is not recommended fo r

use within NHS Scotland for the intrathecal treatment of lymphomatous meningitis. There is limited clinical evidence to support a claim of superior efficacy for liposomal cytara b in e over existing therapy. Effects on symptom improvement and quality of life were not well defined.

The manufacturer did not present a sufficiently robust economic analysis and its justifica t io n o f the treatment’s cost in relation to its health benefits was not sufficient to gain acceptance by

SMC.

NOT RECOMMENDED



09 September 2020


53/97

Product

Manufacturer





Date of NHS Lothian

decision

liposomal daunorubicin/cytarabine

44mg/100mg powder for concentrate for solution for infusion (Vyxeos

®)

Jazz Pharmaceuticals UK Ltd

11.03.19 SMC Report No. 2130

daunorubicin/cytarabine (Vyxeos®) is accepted for use within NHSScotland.

Indication under review: The treatment of adults with newly diagnosed, therapy -re la ted a cu te myeloid leukaemia (AML) or AML with myelodysplasia-related changes.In a randomised p ha se

III study, in adults (aged 60 to 75 years) with high risk AML, liposomal daunorubicin/cyta rab in e improved overall survival when compared with a standard of care regimen.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of liposomal daunorubicin/cytarabine. This advice is contingent upon th e

continuing availability of the PAS in NHSScotland or a list price that is equivalent or lower.



July 2019

liposomal irinotecan hydrochloride trihydrate (as irinotecan sucrosofate salt),

5mg/mL concentrate for solution for infusion (Onivyde

®)

Shire

13.03.17 SMC Report No. 1217/17

NOT RECOMMENDED: Following a full submission assessed under the orphan and end of l ife process, liposomal irinotecan (Onivyde

®) is not recommended for use within NHS Scotland.

Indication under review: Treatment of metastatic adenocarcinoma of the pancreas, in combination with fluorouracil (5-FU) and leucovorin (folinic acid), in adult patients who have

progressed following gemcitabine based therapy. The addition of liposomal irinotecan to 5-FU/folinic acid, compared with 5-FU/folinic acid a lo n e ,

significantly improved overall survival and progression free survival in patients with metastatic adenocarcinoma of the pancreas who had progressed after gemcitabine-based therapy.

The submitting company’s justification of the treatment’s costs in relation to its health b e n e fit s was not sufficient and in addition did not present a sufficiently robust clinical or economic case to

gain acceptance by SMC. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.

NOT RECOMMENDED

lorlatinib 25mg and 100mg film-coated tablets (Lorviqua

®)

Pfizer Limited

09.03.20


Following a full submission assessed under the end of life process, lorlatinib (Lorviqua®) is

accepted for use within NHSScotland on an interim basis subject to ongoing evaluation and

future reassessment. Indication under review: as monotherapy for the treatment of adult patients with anaplastic

lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC) whose dise a se has progressed after:

alectinib or ceritinib as the first ALK tyrosine kinase inhibitor (TKI) therapy; or crizotinib and at least one other ALK TKI

In the relevant subgroup of a non-comparative phase I/II study of previously-treated patients with ALK-positive advanced NSCLC, lorlatinib was associated with an objective response rate of

approximately 40%. This advice applies only in the context of an approved NHSScotland Patient Acce ss Sch eme





August 2020

lutetium (177

Lu) oxodotreotide

370MBq/mL solution for infusion (Lutathera

®)

Advanced Accelerator Applications

08.06.18 SMC Report No. 1337/18

Following a full submission assessed under the orphan medicine process, lutetium (177

Lu)

oxodotreotide (Lutathera®) is accepted for use within NHS Scotland.

Indication under review: for the treatment of unresectable or metastatic, progressive, well

differentiated (G1 and G2), somatostatin receptor positive gastroenteropancreatic neuroendocrine tumours (GEP-NETs) in adults.

In an open-label, phase III study, lutetium (177

Lu) oxodotreotide significantly improved progression-free survival compared with a high dose somatostatin analogue in patients with

progressive midgut neuroendocrine tumours. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.


experts do not wish to add the medicine to the formulary at this time.

There is ongoing work to access this

medicine through national centres

August 2018



09 September 2020


54/97

Product

Manufacturer





Date of NHS Lothian

decision

mercaptopurine 20mg/mL oral

suspension (Xaluprine®)

Nova Laboratories Limited

13.08.12



mercaptopurine 20mg/mL oral suspension (Xaluprine®) is accepted for use within NHS Scotland

for the treatment of acute lymphoblastic leukaemia (ALL) in adults, adolescents and children. Mercaptopurine dosing is governed by cautiously monitoring haematotoxicity. The oral

suspension and tablet formulations are not bioequivalent in terms of peak plasma concentrations and therefore careful haematological monitoring of the patient is advised on switching

formulations. Mercaptopurine oral suspension is more expensive than the tablet formulation.

Included on the Additional List for the

indication in question, where an oral suspension is required.

August 2012

methotrexate 2mg/mL oral solution


As treatment of acute lymphoblastic leukaemia and inflammatory arthritis. Included on the LJF

• For acute lymphoblastic

leukaemia: Additional List.

• For inflammatory arthritis: as first choice, Specialist Initiation.

March 2016

methyl aminolevulinate cream (Metvix) Galderma

0.11.03


methyl aminolevulinate cream (Metvix) is accepted for restricted use within NHS Sco t la n d fo r the treatment of basal cell carcinoma in those patients in whom standard treatment with surgery

or cryotherapy is contraindicated. Its use should be restricted to specialist dermatologists and to superficial lesions where

penetration is most effective.

Added to the Additional List.

October 2005

midostaurin 25mg soft capsules

(Rydapt®)


04.05.18


Following a full submission assessed under the ultra-orphan and end of life medicine p ro ce ss,

midostaurin (Rydapt®) is accepted for use within NHS Scotland.

Indication under review: In combination with standard daunorubicin and cytarabine induction and

high-dose cytarabine consolidation chemotherapy, and for patients in complete response followed by midostaurin single agent maintenance therapy, for adult patients with newly

diagnosed acute myeloid leukaemia (AML) who are FMS-like tyrosine kinase 3 (FLT3) mutation-positive.

In a randomised, double-blind, phase III study of adults (aged <60 years) with FLT3 mutation-positive AML, the addition of midostaurin to standard intensive chemotherapy regime n

resulted in improved overall survival when compared with addition of placebo.



December 2018

midostaurin 25mg soft capsules (Rydapt

®)


09.07.18 SMC Report No. 2100

NON SUBMISSION

NOT RECOMMENDED: midostaurin (Rydapt®) is not recommended for use within NHS

Scotland.

Indication under review:as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasm, or

mast cell leukaemia. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

mifamurtide, 4mg powder for suspensio n for infusion (Mepact

®)

Takeda UK and Ireland Ltd

08.08.11 SMC Report No. 621/10


mifamurtide (Mepact) is accepted for use within NHS Scotland. Indication under review: in combination with post-operative multi-agent chemotherapy for the

treatment of high-grade resectable non-metastatic osteosarcoma after macroscopically complete surgical resection, in children, adolescents and young adults. Safety and effica cy h a ve b e e n

assessed in studies of patients 2 to 30 years of age at initial diagnosis. Mifamurtide has been shown to increase overall survival compared with multi-agent

chemotherapy alone in patients aged up to 30 years with newly-diagnosed resectable osteosarcoma.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of mifamurtide. This SMC advice is contingent upon the continuing

availability of the PAS in NHS Scotland.


September 2011



09 September 2020


55/97

Product

Manufacturer





Date of NHS Lothian

decision

mitotane 500mg tablets (Lysodren®)

Laboratoire HRA Pharma

11.12.06 SMC Report No. 328/06

NOT RECOMMENDED:mitotane (Lysodren®) is not recommended for use within NHS Scotla n d

for the symptomatic treatment of advanced (unresectable, metastatic or relapsed) adrenal cortical carcinoma. The effect of mitotane on non-functional adrenal cortical carcin o ma is n o t

established. Mitotane relieves the symptoms of advanced adrenal cortical carcinoma, but there is insufficien t

evidence to support an increase in survival. The economic case has not been demonstrated. Mitotane should be used only within the context of clinical trials.

NOT RECOMMENDED

mycophenolate mofetil (CellCept ®)

Roche


Management of steroid-dependent nephrotic syndrome in paediatric patients.

Added to the Additional List and

prescribed in accordance with Shared Care Protocol.

Categorised AMBER for paediatrics

under the ADTC ‘Policy for the use of unlicensed (and off-label use) Medicines in

NHS Lothian’.

April 2012

mycophenolic acid (as mycophenolate sodium), 180mg and 360mg film-coated

gastro-resistant tablets (Myfortic®)


07.02.05


mycophenolate sodium (Myfortic®) is accepted for use within NHS Scotland for the proph yla xis

of acute transplant rejection in adult patients receiving allogenic renal transplants in combination

with ciclosporin and corticosteroids. It is restricted to use by transplant specialists as part of an immunosuppressive regimen.

Added to the Additional List. Suitable for shared care.

March 2012

necitumumab (Portrazza®) 800mg

concentrate for solution for infusion Eli Lilly and Company Limited

08.08.16


NOT RECOMMENDED:necitumumab (Portrazza®) is not recommended for use within NHS

Scotland in combination with gemcitabine and cisplatin chemotherapy for the treatment of ad u lt patients with locally advanced or metastatic epidermal growth factor receptor (EGFR)

expressing squamous non-small cell lung cancer who have not received prior chemotherapy fo r this condition.


NOT RECOMMENDED

nelarabine, 5mg/mL solution for infusion

(Atriance®)

GlaxoSmithKline UK

07.04.08


nelarabine (Atriance®) is accepted for restricted use within NHS Scotland for the treatment of

patients with T-cell acute lymphoblastic leukaemia (T-ALL) and T-cell lymphoblastic lymp h o ma (T-LBL) whose disease has not responded to, or has relapsed following, treatment with at le a st

two chemotherapy regimens. It is restricted to patients in whom nelarabine is being used as a treatment to bridge to allogeneic

stem cell transplant and restricted to use by specialists in haemato-oncology. It is not cost-effective when used for palliation.

Added to the Additional List, Specialist

Use only.

November 2009

neratinib (Nerlynx®) 40mg film-coated

tablets Pierre Fabre Limited

10.08.20


neratinib (Nerlynx®) is accepted for use within NHSScotland.

Indication under review:forthe extended adjuvant treatment of adult patients with early-stage hormone receptor positive HER2-overexpressed/amplified breast cancer and who completed

adjuvant trastuzumab-based therapy less than one year ago. In the relevant subgroup of a phase III study neratinib, given less than one year after adjuvant

trastuzumab-based therapy, improved invasive disease-free survival in patients with early-stage hormone receptor positive HER2-overexpressed/amplified breast cancer compared with

placebo. This advice applies only in the context of an approved NHSScotland Patient Access Scheme


Formulary classification not yet decide d –

waiting for information from clinician



09 September 2020


56/97

Product

Manufacturer





Date of NHS Lothian

decision

netupitant/palonosetron 300mg/0.5mg,

hard capsule (Akynzeo®)

Chugai Pharma UK Limited

11.01.16


netupitant/palonosetron (Akynzeo®) is accepted for restricted use within NHS Scotland.

Indication under review: in adults for the prevention of acute and delayed nausea and vomiting associated with highly emetogenic cisplatin-based cancer chemotherapy and moderately

emetogenic cancer chemotherapy. SMC restriction: prevention of acute and delayed nausea and vomiting associated with highly

emetogenic cisplatin-based cancer chemotherapy. In patients receiving a first course of highly emetogenic cisplatin-based chemotherapy, treatment

with netupitant/palonosetron plus dexamethasone resulted in a significantly higher proportion of patients achieving no emesis and no breakthrough medication compared with palonosetron plus

dexamethasone. This advice takes account of the benefits of Patient Access Scheme (PAS) that improves the

cost-effectiveness of netupitant/palonosetron. This advice is contingent upon the continuing availability of the patient access scheme in NHS Scotland or a list price that is equivalent or

lower.



July 2016

nilotinib, 200mg capsules (Tasigna®)


January 2012



NICE MTA 241 states that nilotinib is recommended for the treatment of chronic or accele ra ted phase Philadelphia-chromosome-positive chronic myeloid leukaemia (CML) in adultswhose CML

is resistant to treatment with standard-dose imatinib, or who have imatinib intolerance, and if the manufacturer makes nilotinib available with the discount agreed as part of the p a t ie nt a cce ss

scheme.


March 2009

nilotinib 150mg and 200mg hard capsules (Tasigna

®)


12.03.18 SMC Report No. 1325/18

NON SUBMISSION

NOT RECOMMENDED: nilotinib (Tasigna®) is not recommended for use within NHS Scotland. Indication under review:

• paediatric patients with newly diagnosed Philadelphia chromosome positive ch ro nic myelogenous leukaemia (CML) in the chronic phase

• paediatric patients with Philadelphia chromosome positive CML in chronic phase with resistance or intolerance to prior therapy including imatinib

The holder of the marketing authorisation has not made a submission to SMC regarding this product in this setting. As a result we cannot recommend its use within NHS Scotland.

NOT RECOMMENDED

nilotinib 150mg hard capsules (Tasigna®)


April 2012



NICE MTA 251 advised that nilotinib is recommended as an option for the first-line treatment o f adults with chronic phase Philadelphia-chromosome-positive CML if the manufacturer makes

nilotinib available with the discount agreed as part of the patient access scheme (PAS). NICE MTA 251 was withdrawn in December 2016 but the NICE MTA 251 advice for nilotinib

remains valid in NHSScotland.


November 2011



09 September 2020


57/97

Product

Manufacturer





Date of NHS Lothian

decision

nintedanib 100mg and 150mg soft

capsules (Vargatef®)

Boehringer Ingelheim International GmbH

13.04.15


nintedanib (Vargatef®) is accepted for use within NHS Scotland in combination with docetaxel for

the treatment of adult patients with locally advanced, metastatic or locally recurre n t n o n -sma ll cell lung cancer (NSCLC) of adenocarcinoma tumour histology after first-line chemotherapy.

Addition of nintedanib to second-line treatment of stage IIIb/IV NSCLC with docetaxel significantly increased overall survival in the subgroup patients with adenocarcinoma tumour

histology. This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e

cost effectiveness of nintedanib and is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.




July 2015

niraparib tosylate monohydrate 100mg

hard capsules (Zejula®)

Tesaro UK Limited

13.08.18


Following a full submission assessed under the end of life and orphan medicine process,

niraparib tosylate monohydrate (Zejula®) is accepted for restricted use within NHS Scotland.

Indication under review: As monotherapy for the maintenance treatment of adult p a t ien ts with

platinum-sensitive relapsed high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum-based chemotherapy.

SMC restriction: to patients who do not have a germline BRCA mutation. Niraparib was assessed in a double blind, randomised, placebo controlled phase III study of

patients with high grade serous, recurrent, platinum-sensitive ovarian, fallopian tube or p rima ry peritoneal cancer in which there had been an objective response to the most recen t p la tin u m-

based chemotherapy regimen. Niraparib maintenance was associated with a significantly improved progression free survival when compared with placebo.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of niraparib. This advice is contingent upon the continuing availa bil it y o f


Not routinely available as local

implementation plans are being developed or the ADTC is waiting for further advice

from local clinical experts – decision expected by 19 December 2018.

November 2018

nivolumab 40mg/4mL and 100mg/10mL

vials of concentrate for solution for infusion (Opdivo

®)

Bristol-Myers Squibb Pharmaceuticals Ltd.

11.07.16


nivolumab (Opdivo®) is accepted for use within NHS Scotland for the treatment of locally

advanced or metastatic squamous non-small cell lung cancer (NSCLC) after prior chemotherapy in adults.

Nivolumab, compared with a standard second-line chemotherapy, significantly increased overall survival in patients with locally advanced or metastatic squamous NSCLC who had received

previous therapy including platinum-based doublet chemotherapy. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of nivolumab. This advice is contingent upon the continuing availability o f




October 2016



09 September 2020


58/97

Product

Manufacturer





Date of NHS Lothian

decision

nivolumab, 10mg/mL, concentrate for

solution for infusion (Opdivo®)


08.08.16



nivolumab (Opdivo®) is accepted for restricted use within NHS Scotland as monotherapy for th e

treatment of advanced (unresectable or metastatic) melanoma in adults. SMC restriction: patients previously untreated with ipilimumab.

In a phase III randomised double-blind study, treatment with nivolumab extended overall survival compared with a palliative chemotherapy in patients with previously untreated advanced

melanoma without a BRAF mutation. In an ongoing open label phase III study, tre a tmen t with nivolumab, at the time of primary analysis, extended overall response rate, compared with

investigator’s choice of chemotherapy in patients with advanced melanoma previously t re a te d with an anti-cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) treatment or an anti-CTL A-4

treatment and a BRAF inhibitor. The base-case economic analysis submitted by the company assumed that patients were

treated for a maximum of two years. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of nivolumab. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.


Not included on the LJF because the NHS

Lothian decision is that the medicine does not represent sufficient added benefit to

other comparator medicines to treat the condition in question.

October 2016

nivolumab, 10mg/mL, concentrate for solution for infusion (Opdivo

®)

Bristol-Myers Squibb Pharmaceutical Limited

10.10.16


nivolumab (Opdivo®) is accepted for restricted use within NHS Scotland for the treatment of

locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) after prior

chemotherapy in adults. SMC restriction: treatment with nivolumab is subject to a two-year clinical stopping rule.

Nivolumab, compared with a standard, second-line chemotherapy, significantly increased overall survival in patients with locally advanced or metastatic non-squamous NSCLC who had received

previous therapy including platinum-based doublet chemotherapy. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of nivolumab. This advice is contingent upon the continuing availability o f the PAS in NHS Scotland or a list price that is equivalent or lower.

This advice takes account of views from a Patient and Clinician Engagement (PACE) meet ing.

Routinely available in line with national guidance. Included on the Additional L ist ,


January 2017

nivolumab 10mg/mL concentrate for solution for infusion (Opdivo

®)


07.11.16


nivolumab (Opdivo®)

is accepted for restricted use within NHS Scotland in combination with ipilimumab for the treatment of advanced (unresectable or metastatic) melanoma in adults.

SMC restriction: for the first-line treatment of advanced melanoma In a randomised, double-blind, phase III study of adults with previously untreated advanced

melanoma nivolumab in combination with ipilimumab was associated with a clinically imp o rta n t and statistically significant improvement in progression-free survival when compared with a

single-agent immunotherapy. Overall survival data are immature. The base-case economic analysis submitted by the company assumed that responding patients

were treated for a maximum of 18 months. SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves th e

cost effectiveness of nivolumab and is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.




January 2017



09 September 2020


59/97

Product

Manufacturer





Date of NHS Lothian

decision

nivolumab, 10mg/mL, concentrate for


Bristol-Myers Squibb Pharmaceutical

Limited

12.06.17 SMC Report No. 1188/16

RESUBMISSION

Following a resubmission assessed under the end of life and orphan medicine process

nivolumab (Opdivo®) is accepted for use within NHS Scotland.

Indication under review: As monotherapy for the treatment of advanced renal cell carcinoma

after prior therapy in adults. Nivolumab, compared with a mammalian target of rapamycin (mTOR) inhibitor, significantly

increased overall survival in patients with advanced or metastatic renal cell carcinoma who h a d received one or two previous regimens of anti-angiogenic therapy.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of nivolumab. This advice is contingent upon the continuing availability o f




August 2017

nivolumab 10mg/mL concentrate for


Bristol-Myers Squibb Pharmaceutical

Limited

10.07.17 SMC Report No. 1240/17


Following a full submission assessed under the end of life and ultra-orphan process

nivolumab (Opdivo®) is accepted for use within NHS Scotland.

Indication under review: the treatment of adult patients with relapsed or refractory classical

Hodgkin lymphoma (cHL) after autologous stem cell transplant (ASCT) and treatment with brentuximab vedotin.

In an open-label, single-arm study, a clinically meaningful objective response rate was achieved in patients with relapsed or refractory cHL treated with nivolumab.

SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves th e cost effectiveness of nivolumab and is contingent upon the continuing availability of the PAS in

NHS Scotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.



April 2018

nivolumab, 10mg/mL concentrate for solution for infusion (Opdivo

®)


11.09.17


Following a full submission assessed under the end of life and ultra-orphan medicine process, nivolumab (Opdivo

®) is accepted for restricted use within NHS Scotland.

Indication under review: As monotherapy, for the treatment of squamous cell cancer of the head and neck (SCCHN) in adults progressing on or after platinum-based therapy.

SMC restriction: treatment with nivolumab is subject to a two year clinical stopping rule. A phase III randomised study demonstrated significantly improved overall survival in patients

receiving nivolumab compared with investigator choice of treatment (taxane, folic acid antagonist or epidermal growth factor receptor monoclonal antibody) in adults with SCCHN wh o

had progressed within six months after platinum-based therapy. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of nivolumab. This advice is contingent upon the continuing availability o f the PAS in NHS Scotland or a list price that is equivalent or lower.




May 2018




15.01.18


NOT RECOMMENDED: nivolumabas monotherapy is indicated for the treatment of locally

advanced unresectable or metastatic urothelial carcinoma in adults after failure of prior platinum-containing therapy.

In a single arm, phase II study of patients with metastatic, or surgically unresectable, uro th elia l carcinoma with progressive disease on or after platinum based chemotherapy, tre a tme n t with

nivolumab resulted in an objective response in 20% of patients. The submitting company did not present a sufficiently robust economic and clinical a n a lysis to


meeting.

NOT RECOMMENDED



09 September 2020


60/97

Product

Manufacturer





Date of NHS Lothian

decision



Bristol-Myers Squibb Pharmaceuticals

Limited

09.11.2018 SMC Report No. 2112


nivolumab (Opdivo®) is accepted for use within NHSScotland.

Indication under review: As monotherapy for the adjuvant treatment of adults with me la n o ma with involvement of lymph nodes or metastatic disease who have undergone complete

resection. Adjuvant treatment with nivolumab improved recurrence free survival compared with another

immunotherapy in adults with melanoma with involvement of lymph nodes or metastatic disease who had undergone complete resection.

SMC advice takes account of the benefit of Patient Access Schemes (PAS) that improve the cost effectiveness of nivolumab and is contingent upon the continuing availability of this PAS in

NHSScotland or a list price that is equivalent or lower.



January 2019

nivolumab 10mg/mL concentrate for solution for infusion (Opdivo

®)

Bristol-Myers Squibb Pharmaceuticals Limited

10.06.19


nivolumab (Opdivo®) is accepted for use within NHSScotland.

Indication under review: in combination with ipilimumab for the first-line treatment of adult

patients with intermediate/poor-risk advanced renal cell carcinoma (RCC). Overall survival was significantly longer in the nivolumab plus ipilimumab group compared with a

multiple receptor tyrosine kinase inhibitor in a phase III study in treatment naïve p a tie n ts with intermediate/poor-risk advanced RCC.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of nivolumab in combination with ipilimumab.

Not routinely available as local implementation plans are being developed

or the ADTC is waiting for further advice from local clinical experts – decision

expected by 18 December 2019.

November 2019

obinutuzumab 1,000mg concentrate for

solution for infusion (Gazyvaro®)


08.12.14


obinutuzumab (Gazyvaro®) is accepted for use within NHS Scotland in combination with

chlorambucil, obinutuzumab is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL) and with comorbidities making them unsuitab le

for full dose fludarabine based therapy. The combination of obinutuzumab plus chlorambucil produced a statistically and clinically

significant increase in progression free survival compared with an alkylating agent alo n e o r a n alkylating agent/antibody combination, in older patients with previously untreated CLL wh o h a d

substantial comorbidities.



January 2015

obinutuzumab 1,000mg concentrate for


Roche Products Ltd.

13.03.17


obinutuzumab in combination with bendamustine followed by obinutuzumab maintenance is

indicated for the treatment of patients with follicular lymphoma who did not respond or who progressed during or up to six months after treatment with rituximab or a rituxima b -co n ta in in g

regimen.



May 2017

obinutuzumab, 1,000mg, concentrate fo r solution for infusion (Gazyvaro

®)


15.01.18 SMC Report No. 1286/18

NOT RECOMMENDED: obinutuzumab in combination with chemotherapy, followed by obinutuzumab maintenance therapy in patients achieving a response, for the treatment of

patients with previously untreated advanced follicular lymphoma. In a phase III study, obinutuzumab decreased the risk of disease progressio n co mp a red with

another monoclonal antibody in a subgroup of patients with previously untreated advanced follicular lymphoma.

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently ro b u st e co no mic

analysis to gain acceptance by SMC.

NOT RECOMMENDED



09 September 2020


61/97

Product

Manufacturer





Date of NHS Lothian

decision

obinutuzumab, 1,000mg, concentrate fo r



10.09.18

SMC Report No. 2015 REBSUBMISSION

NOT RECOMMENDED: obinutuzumab (Gazyvaro®) is not recommended for use within

NHSScotland. Indication under review:obinutuzumab in combination with chemotherapy, followed by

obinutuzumab maintenance therapy in patients achieving a response, for the treatment of patients with previously untreated advanced follicular lymphoma.

In a phase III study, obinutuzumab decreased the risk of disease progressio n co mp a red with another monoclonal antibody in a subgroup of patients with previously untreated advanced

follicular lymphoma. The submitting company did not present a sufficiently robust economic analysis to gain

acceptance by SMC.

NOT RECOMMENDED

ofatumumab, 100mg concentrate for solution for infusion (Arzerra

®)

GlaxoSmithKline

09.08.10 SMC Report No. 626/10

NOT RECOMMENDED: ofatumumab (Arzerra®) is not recommended for use within NHS

Scotland. Indication under review: the treatment of chronic lymphocytic leukaemia (CLL) in pat ie n ts wh o are refractory to fludarabine and alemtuzumab.

Interim analysis of a non-randomised, single-arm small study in a subgroup of patients refractory to fludarabine and alemtuzumab found that ofatumumab produced a response rate of 58%.

The manufacturer’s justification of the treatment’s cost in relation to its health benefit s w a s n o t sufficient to gain acceptance by SMC and in addition the manufacturer did not present a

sufficiently robust economic analysis.

NOT RECOMMENDED

ofatumumab 100mg and 1,000mg concentrate for solution for infusion

(Arzerra®)

Novartis

11.05.15


ofatumumab (Arzerra®) is accepted for restricted use within NHS Scotland.

Indication under review: ofatumumab in combination with chlorambucil or bendamustine is

indicated for the treatment of patients with chronic lymphocytic leukaemia (CLL) who h a ve n o t received prior therapy and who are not eligible for fludarabine-based therapy.

SMC restriction: for use in patients who would not be considered for bendamustine therapy a n d who would receive chlorambucil-based therapy.

The combination of ofatumumab plus chlorambucil produced a statistically and clinically significant increase in progression free survival compared with an alkylating agent alone in older

patients with previously untreated CLL who had co-morbidities. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of ofatumumab and it is contingent upon the continuing availability of th e PAS in NHS Scotland or a list price that is equivalent or lower.


Not included because clinicians do not support the formulary inclusion.

July 2015

ofatumumab (Arzerra®) 100mg & 1000mg



10.04.17 SMC Report No. 1237/17

NON SUBMISSION

NOT RECOMMENDED: In the absence of a submission from the holder of the marketing authorisation, ofatumumab (Arzerra

®) is not recommended for use within NHS Scotland.

Indication under review: Treatment of adult patients with relapsed CLL in combination with fludarabine and cyclosphosphamide.


NOT RECOMMENDED



09 September 2020


62/97

Product

Manufacturer





Date of NHS Lothian

decision

olaparib, 50mg, hard capsules

(Lynparza®)

AstraZeneca UK

07.11.16



olaparib (Lynparza®) is accepted for use within NHS Scotland as monotherapy for the

maintenance treatment of adult patients with platinum-sensitive relapsed BRCA-mutated (germline and/or somatic) high grade serous epithelial ovarian, fallopian tube, or primary

peritoneal cancer who are in response (complete response or partial response) to platinum-based chemotherapy.

Olaparib was assessed in a phase II randomised, placebo-controlled study of patients with hig h grade serous, recurrent, platinum-sensitive ovarian, fallopian-tube or primary peritoneal ca n ce r

in which there had been an objective response to the most recent platinum-based chemotherapy regimen. In a pre-planned analysis of the sub-group of patients with BRCA mutatio n , o la p arib

was associated with a significantly improved progression-free survival compared with p la ce b o . An interim analysis of overall survival in the BRCA mutation sub-group (70% maturity)

demonstrated a benefit of more than four months for olaparib over placebo. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of olaparib. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.




question.

January 2017

olaparib, 100mg and 150mg, film-coated

tablets (Lynparza®)

AstraZeneca UK

09.12.19


olaparib (Lynparza®) is accepted for use within NHSScotland.

Indication under review: for the maintenance treatment of adult patients with adva n ced (FIGO stages III and IV) BRCA1/2-mutated (germline and/or somatic) high-grade epithelial ovarian,

fallopian tube or primary peritoneal cancer who are in response (complete or partial) fo llo win g completion of first-line platinum-based chemotherapy.

In a phase III study, olaparib prolonged progression-free survival compared with placebo. This advice applies only in the context of an approved NHSScotland Patient Acce ss Sch eme

(PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a list price that is equivalent or lower.




March 2020

ondansetron 4mg, 8mg orodispersible films (Setofilm

®)

Norgine

11.11.13 SMC Report No.912/13


ondansetron orodispersible films (Setofilm®) are accepted for restricted use within NHS Scotland

for the use:

In adults:

• Prophylaxis of acute nausea and vomiting induced by moderately emetogenic chemotherapy.

• Prophylaxis and treatment of delayed nausea and vomiting induced by moderately to h ig h ly emetogenic chemotherapy.

• Prophylaxis and treatment of acute and delayed nausea and vomiting induced by highly emetogenic radiotherapy.

• Prophylaxis and treatment of post-operative nausea and vomiting (PONV). In paediatric populations:

• Management of chemotherapy-induced nausea and vomiting in children aged ≥6 months.

• Prophylaxis and treatment of post-operative nausea and vomiting (PONV) in children aged ≥4 years.

SMC restriction: ondansetron orodispersible films are restricted to use in patients with an

enhanced risk of aspiration or who experience difficulties in swallowing. Generic preparations of ondansetron are available at a lower cost than the proprietary products.

Included on the Additional List, for the indication in question.

November 2013



09 September 2020


63/97

Product

Manufacturer





Date of NHS Lothian

decision

osimertinib 40mg and 80mg film-coated

tablets (Tagrisso®)


13.02.17

SMC Report No.1214/17 Patient Access Scheme

osimertinib (Tagrisso®) is accepted for restricted use within NHS Scotland for the treatment of

adult patients with locally advanced or metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small-cell lung cancer (NSCLC).

SMC Restriction: in patients who have received previous treatment with an EGFR tyrosine kinase inhibitor.

Osimertinib was associated with an overall response rate of 66% in the pooled analysis o f two phase II single-arm studies of patients with EGFR T790M advanced NSCLC who had re ce ive d

previous treatment with an EGFR tyrosine kinase inhibitor. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of osimertinib. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.




question.

April 2017

osimertinib 40mg and 80mg film-coated tablets (Tagrisso

®)



NOT RECOMMENDED: osimertinib (Tagrisso®) is not recommended for use within

NHSScotland as monotherapy for the first-line treatment of adult patients with locally adva n ce d

or metastatic non-small cell lung cancer (NSCLC) with activating epidermal growth factor receptor (EGFR) mutations.

Osimertinib, compared with two other EGFR tyrosine kinase inhibitors, improved progression -free survival in adults with locally advanced or metastatic NSCLC with activating EGFR

mutations. The submitting company did not present a sufficiently robust economic analysis to gain

acceptance by SMC.

NOT RECOMMENDED

oxaliplatin 50mg, 100mg powder for intravenous infusion (Eloxatin

®)

Sanofi-aventis

07.11.05 SMC Report No. 211/05

oxaliplatin (Eloxatin®) is accepted for use within NHS Scotland, in combination with fluoro u ra cil

and folinic acid, for the adjuvant treatment of stage III (Dukes’ C) colon cancer a f te r co mp le te

resection of the primary tumour. Addition of oxaliplatin to a standard regimen of fluorouracil and folinic acid increase d d ise a se -

free survival in patients who had undergone complete resection of stage III (Dukes’ C) colon cancer. An economic evaluation demonstrated that this is a cost effective treatment op tio n fo r

these patients. Treatment with oxaliplatin (Eloxatin®) should be under the supervision of an

oncologist.


November 2005

oxaliplatin (Eloxitan®)

Sanofi Aventis


Oesophagogastric cancer Added to the Additional List. Categorised RED under the ADTC ‘Po licy


May 2006

oxaliplatin (Eloxitan®)

Sanofi Aventis


Treatment of metastatic or locally advanced inoperable oesophagogastric carcinoma (in combination with epirubicin and capecitabine (EOX))




March 2009

oxaliplatin, 5-fluorouracil and folinic acid


Second-line combination chemotherapy regimen for patients with pancreatic

cancer who have progressed after first–line gemcitabine-based chemotherapy and are of very good performance status.




July 2013



09 September 2020


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Product

Manufacturer





Date of NHS Lothian

decision

oxycodone (OxyNorm®)


11.10.04 SMC Report No. 125/04

oxycodone (OxyNorm®) injection is accepted for restricted use within NHS Scotland only for th e

treatment of moderate to severe pain in patients with cancer. Use of this drug should be restricted to patients who have difficulty in tolerating morphine or

diamorphine therapy. Limited data indicate that it provides analgesia similar to parenteral morphine at similar doses. However, there are no comparative data with diamorphine, the

opioid recommended by Scottish Intercollegiate Guidelines Network (SIGN) for patients with cancer who require parenteral opioids. Oxycodone is more expensive than diamorphine and the

economic case for this product replacing the other products has not been clearly demonstrated. Other indications for this medicine, treatment of moderate to severe post-operative pain and

severe pain requiring the use of strong opioid, have yet to be considered by the Scottish Medicines Consortium. Advice on these indications will be made after the relevant submission s

have been made by the licence holder.

Added to the Formulary for palliative care.

To be initiated by Specialists in patients unable to tolerate morphine or

diamorphine therapy.

November 2004

oxycodone hydrochloride 50mg/mL solution for injection or infusion

(OxyNorm®)


08.11.10


oxycodone hydrochloride 50mg/ml injection (OxyNorm®) is accepted for restricted use within

NHS Scotland.

Indication under review: treatment of moderate to severe pain in patients with cancer SMC restriction: patients who have difficulty in tolerating morphine or diamorphine therap y a n d

who require a high dose of oxycodone delivered via syringe pump which necessitates the d a ily preparation of an additional syringe pump if oxycodone 10mg/mL is used.

No new clinical or pharmacokinetic evidence has been presented for this higher strength formulation. Comparative evidence of analgesia achieved with parenteral administration is

extrapolated from the lower strength 10mg/mL oxycodone formulation compared with morph in e 10mg/mL.

The economic case was made only for patients in a hospice or community setting who require a high dose of oxycodone which necessitates the daily preparation of an additional syringe pump.

Care should be taken to minimise any risk of administration error with the introduction of this increased strength formulation.

Oxycodone 50mg/mL is also licensed for the treatment of moderate to severe post -operative pain and severe pain requiring the use of strong opioid. The manufacturer’s submission related

only to use in moderate to severe pain in patients with cancer therefore SMC cannot recommend the use of oxycodone 50mg/mL injection in the treatment of non-cancer pain.

‘Not preferred’ in Lothian as suitable alternatives exist.

August 2011

paclitaxel (Abraxane®)

AbraxisBioScience Limited

12.04.10 SMC Report No. 556/09

paclitaxel albumin (Abraxane®) is accepted for restricted use within NHS Scotland.

Licensed indication under review: the treatment of metastatic breast cancer in patients who have failed first-line treatment for metastatic disease and for whom standard anthracycline containing

therapy is not indicated. SMC restriction: Use is restricted to patients who would otherwise receive docetaxel or 3 -weekly

solvent-based paclitaxel as second-line treatment for metastatic breast cancer. In one study the overall response rate for paclitaxel albumin was significantly superior to solvent-

based paclitaxel in a subgroup analysis of patients who had previously rece ive d o n e o r mo re lines of therapy for metastatic disease.

The health economic case was only demonstrated for a subset of the licensed indication wh ich is the basis for the SMC restriction.

Note that paclitaxel albumin may have substantially different pharmacological properties compared to other formulations of paclitaxel and is licensed for use in a 3-weekly dosage

schedule.


Use only. September 2010



09 September 2020


65/97

Product

Manufacturer





Date of NHS Lothian

decision

paclitaxel albumin (Abraxane®)

Celgene Ltd.

08.06.15 SMC Report No. 1071/15

NON SUBMISSION

NOT RECOMMENDED: paclitaxel albumin (Abraxane®) is not recommended for use within NHS

Scotland. Indication under review: in combination with carboplatin for the first-line treatment of non-small

cell lung cancer in adult patients who are not candidates for potentially curative surgery and/or radiation therapy.


NOT RECOMMENDED

paclitaxel formulated as albumin bound

nanoparticles 5mg/mL powder for suspension for infusion (Abraxane

®)

Celgene Ltd.

09.02.15 SMC Report No. 968/14

RESUBMISSION

paclitaxel albumin (Abraxane®) is accepted for use within NHS Scotland in combination with

gemcitabine for the first-line treatment of adult patients with metastatic adenocarcinoma o f th e pancreas.

In a randomised, phase III, open-label study paclitaxel albumin plus gemcitabine treatment improved median overall survival by 1.8 months compared with gemcitabine alone.




April 2015

paclitaxel

April 2016 NICE MTA 389

NICE MTA 389 Topotecan, pegylated liposomal doxorubicin hydrochloride, paclitaxel, trabectedin and gemcitabine for treating recurrent ovarian cancer

Paclitaxel in combination with platinum or as monotherapy is recommended within its marketing authorisation as an option for treating recurrent ovarian cancer.



March 2017

palbociclib 75mg, 100mg and 125mg

hard capsules (Ibrance®)

Pfizer Limited.

11.12.17 SMC Report No. 1276/17


palbociclib (Ibrance®) is accepted for restricted use within NHS Scotland.

Indication under review: treatment of hormone receptor (HR)-positive, human epidermal gro wth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer:

- in combination with an aromatase inhibitor; - in combination with fulvestrant in women who have received prior endocrine therapy.

In pre- or peri-menopausal women, the endocrine therapy should be combined with a luteinising hormone-releasing hormone (LHRH) agonist.

SMC restriction: in combination with an aromatase inhibitor for first-line treatment of HR-positive HER2-negative locally advanced or metastatic breast cancer.

In an open label phase II study and a double-blind, placebo-controlled phase III study, palbociclib in combination with letrozole increased progression-free survival when compared

with letrozole alone in patients with oestrogen receptor-positive, HER2-negative advanced breast cancer.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of palbociclib. This advice is contingent upon the continuing availability o f


meeting.



July 2018



09 September 2020


66/97

Product

Manufacturer





Date of NHS Lothian

decision

palbociclib 75mg, 100mg and 125mg

hard capsules (Ibrance®)

Pfizer Limited.

08.07.19


palbociclib (Ibrance®) is accepted for use within NHSScotland.

Indication under review: for the treatment of hormone receptor (HR)-positive, human epide rma l growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer:

- in combination with an aromatase inhibitor; - in combination with fulvestrant in women who have received prior endocrine therapy.

In pre- or perimenopausal women, the endocrine therapy should be combined with a luteiniz in g hormone-releasing hormone (LHRH) agonist.

This submission relates to use in combination with fulvestrant in women who have received prior endocrine therapy.

In a phase III study palbociclib plus fulvestrant, compared with fulvestrant, prolonged progression-free survival in women with HR-positive HER2-negative locally advanced or

metastatic breast cancer who had received prior endocrine therapy. This SMC advice takes account of the benefits of Patient Access Schemes (PAS) that imp ro ve

the cost-effectiveness of palbociclib and fulvestrant. This advice is contingent upon the continuing availability of these PAS in NHSScotland or list prices that are equivalent or lower.


SMC has previously accepted palbociclib for restricted use in combination with an aromatase inhibitor for first-line treatment of HR-positive HER2-negative locally advanced or metastatic

breast cancer (SMC 1276/17). This advice remains valid.



November 2019

palifermin (Kepivance®)

Amgen Ltd

08.05.06 SMC Report No. 272/06

NON SUBMISSION

NOT RECOMMENDED: palifermin (Kepivance®) is not recommended for use within NHS

Scotland for the treatment of oral mucositis in bone marrow transplantation. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

palonosetron 250micrograms solution fo r injection (Aloxi

®)

Cambridge Laboratories

07.11.05 SMC Report No. 208/05

palonosetron (Aloxi®) is accepted for use within NHS Scotland for the prevention of acute

nausea and vomiting associated with highly emetogenic cancer chemotherapy and the

prevention of nausea and vomiting associated with moderately emetogenic cancer chemotherapy.

It is as effective as other 5HT3 antagonists in preventing emesis when given as a single intravenous injection following highly emetogenic chemotherapy (HEC) in the acute pha se a n d

moderately emetogenic chemotherapy (MEC) in the acute and delayed phases post-chemotherapy.


July 2008

palonosetron 500microgram soft capsules

(Aloxi®)

Sinclair IS Pharma

11.02.13



palonosetron soft capsules (Aloxi®) is accepted for use within NHS Scotland for p re ve n t io n o f

nausea and vomiting associated with moderately emetogenic cancer chemotherapy in adults. At recommended licensed doses the soft capsule formulation has been shown to b e clin ica lly

non-inferior to the intravenous formulation and is cost neutral. SMC has previously accepted palonosetron intravenous injection for the prevention o f n a u se a

and vomiting associated with moderately emetogenic cancer chemotherapy.

Includedon the Additional List for the

indication in question.

January 2013



09 September 2020


67/97

Product

Manufacturer





Date of NHS Lothian

decision

palonosetron, 250 micrograms solution

for injection (Aloxi®)

Chugai Pharma UK Limited

10.08.15



palonosetron (Aloxi®) is accepted for use within NHS Scotland.

Indication under review: prevention of acute nausea and vomiting associated with highly emetogenic cancer chemotherapy and prevention of nausea and vomiting associated with

moderately emetogenic cancer chemotherapy, in paediatric patients 1 month of age and older. A phase III double blind study demonstrated non-inferiority of palonosetron to another 5-HT3

antagonist in paediatric patients.


Use only.

September 2015

panitumumab 20mg/mL concentrate for solution for infusion (Vectibix)

Amgen Ltd

January 2012 NICE MTA 242 supersedes SMC Report

No. 486/08

NOT RECOMMENDED: NICE MTA 242 Panitumumab monotherapy is not recommended for the treatment of people with metastatic colorectal cancer that has progressed after first-line

chemotherapy.

NOT RECOMMENDED

panitumumab (Vectibix®) 20 mg/mL


Amgen Ltd

April 2017 NICE MTA 439 supersedes SMC Report

No. 769/12 Patient Access Scheme

NICE MTA 439 Cetuximab and panitumumab for previously untreated metastatic colorectal cancer. Panitumumab is recommended, within its marketing authorisation, as an option for

previously untreated RAS wild-type metastatic colorectal cancer in adults in combination with: FOLFOX, or FOLFIRI.



May 2017

panitumumab (Vectibix®)

Amgen Ltd

April 2017



NICE MTA 439 Cetuximab and panitumumab for previously untreated metastatic colorectal cancer. Panitumumab is recommended, within its marketing authorisation, as an option for

previously untreated RAS wild-type metastatic colorectal cancer in adults in combination with: FOLFOX, orFOLFIRI.

SMC or NICE MTA 439 Recommendations

1.1 Cetuximab is recommended, within its marketing authorisation, as an option for p re vio u sly untreated epidermal growth factor receptor (EGFR)-expressing, RAS wild-type metastatic

colorectal cancer in adults in combination with: • 5 fluorouracil, folinic acid and oxaliplatin (FOLFOX) or

• 5 fluorouracil, folinic acid and irinotecan (FOLFIRI). 1.2 Panitumumab is recommended, within its marketing authorisation, as an option for

previously untreated RAS wild-type metastatic colorectal cancer in adults in combination with: • FOLFOX or

• FOLFIRI. 1.3 The drugs are recommended only when the companies provide them with the discount

agreed in the patient access scheme (for panitumumab) or commercial access agree me n t (fo r cetuximab).

Routinely available in line with local guidance. Included on the Additional L ist ,


July 2020



09 September 2020


68/97

Product

Manufacturer





Date of NHS Lothian

decision

panobinostat, 10mg, 15mg and 20mg

hard capsules (Farydak®)

Novartis Europharm Limited

08.02.16


panobinostat (Farydak®) is accepted for use within NHS Scotland.

Indication under review: In combination with bortezomib and dexamethasone, for the treatme n t of adult patients with relapsed and/or refractory multiple myeloma who have rece ive d a t le a st

two prior regimens including bortezomib and an immunomodulatory agent. In patients with relapsed or relapsed and refractory multiple myeloma, panobinostat in

combination with bortezomib plus dexamethasone was associated with a significant b e n ef it in progression-free survival (PFS) compared with bortezomib plus dexamethasone. The treatme n t

effect of the panobinostat containing regimen on PFS was greater in the subgroup of p a t ie n ts’ representative of the licensed indication.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of panobinostat. This advice is contingent upon the continuing availabilit y

of the PAS in NHS Scotland or a list price that is equivalent or lower.



July 2016

pasireotide (as pamoate), 20mg, 40mg 60mg powder and solvent for suspension

for injection (Signifor®)

Novartis Pharmaceuticals UK Ltd.

07.09.15


pasireotide (as pamoate) (Signifor®) is accepted for use within NHS Scotland.

Indication under review: Treatment of adult patients with acromegaly for whom surgery is not an

option or has not been curative and who are inadequately controlled on treatment with another somatostatin analogue.

Pasireotide administered every four weeks was significantly superior to an active control group (comprising other somatostatin analogues administered monthly) for the primary endpoint of

biochemical control, in patients with inadequately controlled acromegaly following treatment with a somatostatin analogue for at least six months.


Includedon the LJF as a prescribing n o te for the indication in question.

October 2015

pazopanib 200mg, 400mg film-coated

tablets (Votrient®)

GlaxoSmithKline UK

07.03.11


pazopanib (Votrient®) is accepted for restricted use within NHS Scotland.

Indication under review: First-line treatment of advanced renal cell carcinoma (RCC) and for patients who have received prior cytokine therapy for advanced disease.

SMC restriction: use is restricted to the first-line treatment of advanced RCC. Pazopanib was superior to placebo for the primary endpoint, progression f ree survival, in the

whole population and the treatment naïve and cytokine pre-treated sub-groups. An indirect comparison demonstrated that pazopanib had similar efficacy to the main comparator.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of pazopanib. This SMC advice is contingent upon the continuing

availability of the Patient Access Scheme in NHS Scotland.


Use only.

September 2011

pazopanib 200mg, 400mg film-coated

tablets (Votrient®)

GlaxoSmithKline UK

10.12.12


NOT RECOMMENDED: pazopanib (Votrient®) is not recommended for use within NHS Scotland

for the treatment of adult patients with selective subtypes of advanced soft tissue sarcoma (STS) who have received prior chemotherapy for metastatic disease or who have progressed within 12

months after (neo) adjuvant therapy. Efficacy and safety has only been established in ce rta in STS histological tumour subtypes.

In a pivotal study, pazopanib significantly improved progression-free survival compared with placebo in adult patients with selective subtypes of advanced STS. However there was no

significant improvement in overall survival. The submitting company's justification of the treatment's cost in relation to its health benefits

was not sufficient to gain acceptance by SMC, and in addition the submitt ing compan y d id n o t present a sufficiently robust economic analysis to gain acceptance by SMC.

NOT RECOMMENDED

PCV – procarbazine 50mg capsules,

lomustine (CCNU) “medac” 40mg capsules and vincristine sulphate 1mg/mL

injection vials / pre-filled infusion


Adjuvant treatment for patients with grade II glioma following radiotherapy.





April 2017



09 September 2020


69/97

Product

Manufacturer





Date of NHS Lothian

decision

pegaspargase (Oncaspar®) 750U/mL

solution for injection/infusion Baxalta

07.11.16



pegaspargase (Oncaspar®) is accepted for use within NHS Scotland as a component of

antineoplastic combination therapy in acute lymphoblastic leukaemia (ALL) in paediatric patients from birth to 18 years, and adult patients.

Pegaspargase (Oncaspar®) has been used in NHS Scotland as an unlicensed medicine fo r th e

treatment of ALL in children and adults; it has now been granted a product licence.

Included on the LJF as first choice,


November 2016

pegylated liposomal doxorubicin hydrochloride (PLDH) (Caelyx

®) in

combination with carboplatin Janssen-Cilag

April 2016

NICE MTA 389

NICE (Multiple) Technology Appraisal Guidance No 389 ‘Topotecan, pegylated liposomal doxorubicin hydrochloride, paclitaxel, trabectedin and gemcitabine for treating recurrent ovarian

cancer’. Pegylated liposomal doxorubicin hydrochloride (PLDH) as monotherapy is recommended within

its marketing authorisation as an option for treating recurrent ovarian cancer. PLDH in combination with platinum is recommended as an option for treating recurrent ovarian

cancer. The Appraisal Committee was unable to make recommendations on the use of these

technologies for treating platinum-sensitive ovarian cancer beyond the first recurrence. The recommendations replace the recommendations in NICE MTA 91 relating to the use of

paclitaxel, pegylated liposomal doxorubicin hydrochloride and topotecan for second -line or subsequent treatment of advanced ovarian cancer.


for Specialist Use only, for the indication in question.

January 2017

pegylated liposomal doxorubicin

(Caelyx®)

Schering-Plough Ltd

09.01.04


NOT RECOMMENDED: This pegylated liposomal formulation of doxorubicin hydrochloride is

now licensed as monotherapy for the treatment of metastatic breast cancer where th e re is a n increased cardiac risk. An inconclusive study has suggested that it was not inferior to

conventional doxorubicin in terms of progression-free survival. It was less cardiotoxic than conventional doxorubicin, but was associated with other troublesome adverse events,

particularly palmar-plantar erythrodysesthesia. The product is significantly more expensive than the standard preparation and its cost effectiveness in managing metastatic breast ca n cer h a s

not been addressed by the company in their submission.

NOT RECOMMENDED

pegylated liposomal doxorubicin, 2mg/mL

concentrate for solution for infusion (Caelyx

®)

Schering Plough

13.07.09 SMC Report No. 503/08

RESUBMISSION

NOT RECOMMENDED: pegylated liposomal doxorubicin (Caelyx®) is not recommended for use

within NHS Scotland in combination with bortezomib for the treatment of progressive multiple myeloma in patients who have received at least one prior therapy and who have already

undergone or are unsuitable for bone marrow transplant. Results from an interim analysis showed that pegylated liposomal doxorubicin plus bortezo mib

significantly increased the time to disease progression compared to bortezomib mon o the ra py. At the time of the interim analysis only 31% of patients in the combination arm had reached th e

primary endpoint. The manufacturer did not present a sufficiently robust economic analysis to gain acceptance b y

SMC.

NOT RECOMMENDED



09 September 2020


70/97

Product

Manufacturer





Date of NHS Lothian

decision

pembrolizumab 50mg powder for

concentrate for solution for infusion (Keytruda

®)


09.11.15 SMC Report No. 1086/15


pembrolizumab (Keytruda®) is accepted for use within NHS Scotland as monotherapy for the

treatment of advanced (unresectable or metastatic) melanoma in adults. This submission relates to use in adults previously untreated with ipilimumab.

In a phase III randomised open-label study, treatment with pembrolizumab (at unlicensed doses) extended median progression free survival and overall survival compared with other immune

therapy in patients with advanced melanoma previously untreated with ipilimumab. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of pembrolizumab. This advice is contingent upon the continuing availability of the patient access scheme in NHS Scotland or a list price that is equivalent or

lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting. SMC has also assessed pembrolizumab as monotherapy for the treatment of advanced

(unresectable or metastatic) melanoma in adults previously treated with ipilimumab and has advised that it is not recommended for use within NHS Scotland in this setting (SMC

No.1087/15).



April 2016

pembrolizumab 50mg powder for concentrate for solution for infusion

(Keytruda®)


12.12.16


NOT RECOMMENDED: pembrolizumab (Keytruda®) is not recommended for use within NHS

Scotland as monotherapy for the treatment of advanced (unresectable or metastatic) melanoma

in adults. This submission relates to use in adults previously treated with ipilimumab. In a phase II randomised study, pembrolizumab improved progression free survival co mp a re d

with chemotherapy in patients with advanced melanoma previously treated with ipilimumab and, if BRAF V600 mutant-positive, a BRAF or MEK inhibitor.

The submitting company did not present a sufficiently robust economic analysis and in addit io n its justification of the treatment’s cost in relation to its health benefits was not sufficien t to g a in

acceptance by SMC. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.

NOT RECOMMENDED


concentrate for solution for infusion (Keytruda

®)


16.01.17 SMC Report No. 1204/17


pembrolizumab (Keytruda®) is accepted for restricted use within NHS Scotland for the treatment

of locally advanced or metastatic non-small cell lung carcinoma (NSCLC) in adults whose tumours express programmed death ligand 1 (PD-L1) and who have received at least one p rio r

chemotherapy regimen. SMC restriction: treatment with pembrolizumab is subject to a two-year clinical stopping rule.

Pembrolizumab, compared with a standard taxane monotherapy, significantly improved o ve ra ll survival in adults with advanced NSCLC tumours that express PD-L1 and have progressed after

platinum-doublet chemotherapy. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of pembrolizumab. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.





March 2017



09 September 2020


71/97

Product

Manufacturer





Date of NHS Lothian

decision


concentrate for solution for infusion and 25mg/mL concentrate for solution for

infusion (Keytruda®)


07.08.17


pembrolizumab (Keytruda®) is accepted for restricted use within NHS Scotland.

Indication under review: As monotherapy for the first-line treatment of metastatic non-small cell lung carcinoma (NSCLC) in adults whose tumours express

programmed death ligand 1 (PD-L1) with a ≥50% tumour proportion score (TPS) with no epidermal growth factor receptor (EGFR) or anaplastic lymphoma kinase

(ALK) positive tumour mutations. SMC restriction: treatment with pembrolizumab is subject to a two-year clinical stopping rule.

In a randomised, open-label, phase III study, treatment with pembrolizumab provided an additional 4.3 months of progression free survival compared to standard of care.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of pembrolizumab. This advice is contingent upon the continuing

availability of the PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician

Engagement (PACE) meeting.



December 2019

pembrolizumab 25mg/mL concentrate for solution for infusion and 50mg powder for

concentrate for solution for infusion (Keytruda®)


12.02.18 SMC Report No. 1291/18



Indication under review: as monotherapy for the treatment of locally advanced or metastatic

urothelial carcinoma in adults who have received prior platinum-containing chemotherapy. SMC restriction: treatment with pembrolizumab is subject to a two-year clinical stopping rule.

In a phase III study of patients with measurable urothelial carcinoma with progressive d ise a se on or after platinum based chemotherapy, treatment with pembrolizumab was associated with a

statistically significant improvement in overall survival when compared with investigator’s choice of single agent chemotherapy.


availability of the PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.



May 2018

pembrolizumab (Keytruda®) 50mg

powder for concentrate for solution for infusion and 25mg/mL concentrate for

solution for infusion Merck Sharp and Dohme Ltd

12.03.18



Indication under review: As monotherapy for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma who have failed autologous stem cell transplant and

brentuximab vedotin, or who are transplant-ineligible and have failed brentuximab vedotin. SMC restriction: treatment with pembrolizumab is subject to a two-year clinical stopping rule.

In a phase II study, pembrolizumab was associated with a clinically meaningful overall response rate in adults with classical Hodgkin lymphoma who had failed autologous stem cell tra n sp la n t

and brentuximab vedotin, or who were transplant-ineligible and had failed brentuximab vedotin. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of pembrolizumab. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting

Not routinely available as local experts d o

not wish to add the medicine to the formulary at this time.

April 2018



09 September 2020


72/97

Product

Manufacturer





Date of NHS Lothian

decision

pembrolizumab 25mg/mL concentrate for

solution for infusion and 50mg powder for concentrate for solution for infusion

(Keytruda®)


10.09.18

SMC ReportNo. 1339/18

NOT RECOMMENDED: Following a full submission considered under the end of life and orphan

equivalent process, pembrolizumab (Keytruda®) is not recommended for use within NHS

Scotland.

Indication under review: as monotherapy, for the treatment of locally advanced or metastat ic urothelial carcinoma in adults who are not eligible for cisplatin-containing chemotherapy and

whose tumours express PD-L1 with a combined positive score (CPS)≥10. In an open-label, non-comparative phase II study of adults with advanced / metastatic urothelia l

cancer who had no previous treatment for advanced / metastatic disease and who were ineligible for first-line cisplatin-based therapy, treatment with pembrolizumab was associated

with an objective response in 47% of patients with strongly positive PD-L1 expression (CPS≥10). The submitting company’s justification of the treatment’s cost in relation to its health benefits

was not sufficient and in addition the company did not present a sufficiently robust clin ica l a n d economic analysis to gain acceptance by SMC.


NOT RECOMMENDED



(Keytruda®)


10.12.18


NOT RECOMMENDED: In the absence of a submission from the holder of the marketing

authorisation pembrolizumab (Keytruda®) is not recommended for use within NHSScotland.

Indication under review: As monotherapy for the treatment of recurrent or metastatic head and

neck squamous cell carcinoma in adults whose tumours express PD-L1 with a ≥50% TPS and progressing on or after platinum-containing chemotherapy.


NOT RECOMMENDED



(Keytruda®)

Merck Sharp & Dohme UK Ltd

13.05.19

SMC Report No. 2144

Following a full submission considered under the orphan equivalent process

pembrolizumab (Keytruda®) is accepted for use within NHSScotland.

Indication under review: As monotherapy for the adjuvant treatment of adults with Stage III

melanoma and lymph node involvement who have undergone complete resection. Recurrence-free survival was significantly longer in the pembrolizumab group compared with

placebo in a phase III study of adult patients with completely resected, stage III melanoma with lymph node involvement.


availability of the PAS in NHSScotland or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting .


experts do not wish to add the medicine at this time or there is a local preference fo r

alternative medicines.

July 2019



09 September 2020


73/97

Product

Manufacturer





Date of NHS Lothian

decision



(Keytruda®)


09.09.19


Following a full submission assessed under the end of life medicine process, pembrolizumab

(Keytruda®) is accepted for restricted use within NHSScotland.

Indication under review: In combination with carboplatin and either paclitaxel or nab-paclita xel,

for the first-line treatment of metastatic squamous non-small cell lung cancer (NSCLC) in adults. SMC restriction: in combination with carboplatin and paclitaxel in patients whose tumours

express programmed death ligand 1 (PD-L1) with a <50% tumour proportion score (TPS), o r in those whom it has not been possible to evaluate PD-L1 TPS. Treatment with pembrolizuma b is

subject to a two-year clinical stopping rule. Pembrolizumab in combination with platinum based doublet chemotherapy was associated with

a progression-free survival and overall survival benefit over platinum based doublet chemotherapy in patients with treatment naïve metastatic squamous NSCLC.


availability of the PAS in NHSScotland or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.



November 2019



(Keytruda®)


07.10.19



pembrolizumab (Keytruda®) is accepted for restricted use within NHSScotland.

Indication under review: in combination with pemetrexed and platinum chemothe ra py, fo r th e first-line treatment of metastatic non-squamous non-small cell lung carcinoma (NSCLC) in adults

whose tumours have no EGFR or ALK positive mutations. SMC restriction: in patients whose tumours express programmed death ligand 1 (PD-L1) with a

<50% tumour proportion score (TPS), or in those whom it has not been possible to evaluate PD-L1 TPS. Treatment with pembrolizumab is subject to a two-year clinical stopping rule.

The addition of pembrolizumab to pemetrexed and platinum chemotherapy significantly improved progression-free survival and overall survival in patients with metastatic non-

squamous NSCLC with no EGFR or ALK mutations. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of pembrolizumab. This advice is contingent upon the continuing availability of the PAS in NHSScotland or a list price that is equivalent or lower.




December 2019



(Keytruda®)

Merck, Sharpe and Dohme Limited

07.09.20 SMC Report No. 2247



Indication under review: in combination with axitinib, for the first-line treatment of advanced renal cell carcinoma in adults.

SMC restriction: treatment with pembrolizumab is subject to a two-year clinical stopping rule. In an open-label, phase III study, first-line treatment with pembrolizumab plus axitinib

significantly improved progression-free and overall survival in adults with advanced renal cell carcinoma compared with a vascular endothelial growth factor (VEGF)-targeting tyrosine-kinase

inhibitor (TKI). This advice applies only in the context of approved NHSScotland Patient Access Scheme (PAS)

arrangements delivering the cost-effectiveness results upon which the decision was based, or PAS/ list prices that are equivalent or lower.






09 September 2020


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(Keytruda®)

Merck, Sharpe and Dohme Limited

07.09.20 SMC Report No. 2257



Indication under review: as monotherapy or in combination with platinum and fluorouracil chemotherapy, for the first-line treatment of metastatic or unresectable recurrent head and neck

squamous cell carcinoma (HNSCC) in adults whose tumours express programmed cell death ligand-1 (PD-L1) with a combined positive score (CPS)≥1.

SMC restriction: treatment with pembrolizumab is subject to a two-year clinical stopping rule. Overall survival was longer in patients who received pembrolizumab as monotherapy or in

combination with chemotherapy compared with a monoclonal antibody plus chemotherapy in a phase III study in patients with untreated, locally incurable, recurrent or metastatic HNSCC with

PD-L1 CPS≥1. This advice applies only in the context of an approved NHSScotland Patient Access Scheme





pemetrexed (Alimta®)

Eli Lilly and Company Limited

08.05.06


NOT RECOMMENDED: pemetrexed (Alimta®) is not recommended for use within NHS Scotland

as monotherapy for the treatment of patients with locally advanced or metastatic non-small ce ll

lung cancer after prior chemotherapy. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

pemetrexed, 500mg vial of powder for

solution for intravenous infusion (Alimta®)


08.09.08


ND RESUBMISSION

pemetrexed (Alimta®) is accepted for restricted use within NHS Scotland for monotherapy for the

second-line treatment of patients with locally advanced or metastatic non-small cell lung can ce r (NSCLC) other than predominantly squamous cell histology.

It is restricted to use in patients with good performance status who would otherwise be e lig ib le for treatment with docetaxel.

In a retrospective unplanned sub-group analysis of a study comparing pemetrexed with anothe r agent used in the second line treatment of NSCLC, treatment with pemetrexed re su lte d in a n

additional median survival of 1.3 months in patients with a non-squamous histology.


Use only.

July 2009

pemetrexed, 100mg, 500mg powder for

concentrate for solution for infusion (Alimta

®)


08.02.10 SMC Report No. 531/09

2nd

RESUBMISSION

pemetrexed (Alimta®) is accepted for restricted use within NHS Scotland in combination with

cisplatin for the first line treatment of patients with locally advanced or metastatic non-small ce ll lung cancer (NSCLC) other than predominantly squamous cell histology.

It is restricted to patients in whom histology has been confirmed as adenocarcinoma or large cell carcinoma.

In a planned subgroup analysis of a study comparing pemetrexed plus cisplatin with another platinum-based combination regimen, treatment with pemetrexed plus cisplatin re su lte d in a n

improvement in median survival in patients with a non-squamous (adenocarcinoma p lu s la rg e cell carcinoma) histology.

Add to the Additional List, for Specialist

Use only as first-line treatment

July 2010

pemetrexed 500mg infusion (Alimta®)

Eli Lilly

05.08.05 SMC Report No. 192/05

pemetrexed (Alimta) in combination with cisplatin is accepted for restricted use within NHS Scotland for the treatment of chemotherapy-naïve patients with stage III/IV unresectable

malignant pleural mesothelioma. Pemetrexed in combination with cisplatin prolonged survival compared with cisplatin alone in

patients with unresectable malignant pleural mesothelioma. Pemetrexed is the first licensed agent for the treatment of malignant pleural mesothelioma.

Pemetrexed is also indicated as monotherapy for the treatment of patients with locally advanced or metastatic non-small cell lung cancer after prior chemotherapy. SMC has not yet receive d a

submission for this indication and therefore cannot currently recommend its use.


Use only.

August 2005



09 September 2020


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pemetrexed, 100mg, 500mg powder for


®)

Eli Lilly

11.10.10 SMC Report No. 642/10

NOT RECOMMENDED: pemetrexed (Alimta®) is not recommended for use within NHS

Scotland. Indication under review: monotherapy for the maintenance treatment of locally advanced or

metastatic non-small cell lung cancer (NSCLC) other than predominantly squamous cell histology in patients whose disease has not progressed immediately following platin um -b a se d

chemotherapy. First-line treatment should be a platinum doublet with gemcitabine, paclitaxel o r docetaxel.

In a sub-group analysis of patients with non-squamous NSCLC, progression free su rviva l a n d overall survival (secondary endpoint) were significantly longer for pemetrexed plus best

supportive care (BSC) compared to placebo plus BSC. However, the manufacturer did not present a sufficiently robust economic case and their

justification of the treatment's cost in relation to its health benefits was not sufficient to gain acceptance by SMC.

NOT RECOMMENDED

pemetrexed, 100mg & 500mg, powder for


®)


08.12.14 SMC Report No. 770/12

pemetrexed (Alimta®) is accepted for use within NHS Scotland as monotherapy for the

maintenance treatment of locally advanced or metastatic non-small cell lung cancer oth e r th a n predominantly squamous cell histology in patients whose disease has not progressed

immediately following platinum-based chemotherapy. In patients with locally advanced or metastatic non-squamous non-small cell lung cancer,

maintenance treatment with pemetrexed, following completion of first -line platinum-based chemotherapy, was associated with prolonged overall survival and progression -free survival

when compared with placebo. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.



January 2015

pertuzumab 30mg/mL concentrate for solution for infusion (Perjeta

®)


07.12.18

SMC Report No. 2120 3

rd RESUBMISSION

Following a third resubmission assessed under the orphan equivalent process, pertuzumab (Perjeta

®) is accepted for use within NHSScotland.

Indication under review: In combination with trastuzumab and docetaxel, in adult pa tie n ts with HER2 positive metastatic or locally recurrent unresectable breast cancer, who have not received

previous anti HER2 therapy or chemotherapy for their metastatic d isease. Addition of pertuzumab to current first-line treatment, trastuzumab plus docetaxel, signif ica nt ly

increased progression-free and overall survival for women with HER2-positive metastatic or locally recurrent unresectable breast cancer.

This SMC advice takes account of the benefit of Patient Access Schemes (PAS) that imp ro ve s the cost effectiveness of pertuzumab and trastuzumab IV (Herceptin®). This advice is

contingent upon the continuing availability of these PAS in NHSScotland or list price s th a t a re equivalent or lower.


Routinely available in line with national guidance. Added to the Additional List fo r


April 2019

pertuzumab 420mg concentrate for solution for infusion (Perjeta

®)


09.11.18


nd RESUBMISSION

Following a second resubmission assessed under the orphan medicine process, p e rtuzu ma b (Perjeta


Indication under review: for use in combination with trastuzumab and chemotherapy in the neoadjuvant treatment of adult patients with HER2-positive, locally advanced, inflammato ry, o r

early stage breast cancer at high risk of recurrence. In a phase II study conducted in women with locally advanced, inflammatory, or early HER2 -

positive breast cancer, in the neoadjuvant setting, the addition of pertuzumab to t ra stu zuma b plus chemotherapy resulted in a significantly higher proportion of patients achieving pathological

complete response in the breast. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.

Routinely available in line with national guidance.Included on the Additional L ist ,

for Specialist Use only, for the indication in question.

January 2019



09 September 2020


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pertuzumab 420mg concentrate for

solution for infusion (Perjeta®)

Roche Products Ltd

07.10.19 SMC Report No. 2197

NOT RECOMMENDED:following a full submission considered under the orphan equivalent

process, pertuzumab (Perjeta®) is not recommended for use within NHSScotland.

Indication under review: for use in combination with trastuzumab and chemotherapy in the

adjuvant treatment of adult patients with HER2-positive early breast cancer at high risk of recurrence.

The addition of pertuzumab to trastuzumab and chemotherapy improved invasive disease-f re e survival in patients with HER2-positive early breast cancer at high risk of recurrence. Overall

survival data are immature. The submitting company did not present a sufficiently robust clinical or economic analysis to


meeting.

NOT RECOMMENDED

pertuzumab 420mg concentrate for solution for infusion (Perjeta

®)


07.09.20


rd RESUBMISSION


Following a resubmission assessed under the orphan medicine process pertuzumab (Perjeta

®) is accepted for restricted use within NHSScotland.

Indication under review: for use in combination with trastuzumab and chemotherapy in the adjuvant treatment of adult patients with HER2-positive early breast cancer at high risk of

recurrence. SMC restriction: for use in patients with lymph node-positive disease.

The addition of pertuzumab to trastuzumab and chemotherapy improved invasive disease-free survival in patients with HER2-positive early breast cancer at high risk of recurrence.

This advice applies only in the context of approved NHSScotland Patient Access Scheme (PAS) arrangements delivering the cost-effectiveness results upon which the decision was based, or

PAS/ list prices that are equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.

Formulary classification not yet decided – waiting for information from clinician.

pixantrone (Pixuvri®) 29 mg power for


CTI Life Sciences Ltd

07.02.16 SMC Report No. 1138/16

NON SUBMISSION

NOT RECOMMENDED: pixantrone (Pixuvri®) is not recommended for use within NHS Scotland.

Indication under review:As monotherapy for the treatment of adult patients with multiply relapsed

or refractory aggressive Non Hodgkin B-cell Lymphomas. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

plerixafor, 20mg/ml solution for injection

(Mozobil®)

Genzyme Therapeutics Ltd.

18.01.10


plerixafor (Mozobil®) is accepted for use within NHS Scotland in combination with G-CSF to

enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with lymphoma and multiple myeloma who se

cells mobilise poorly. Significantly more patients treated with plerixafor than with placebo achieved their target

collection of CD 34+ cells required for autologous stem cell transplantation with subsequent sustained engraftment.


Use only.

August 2010



09 September 2020


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Product

Manufacturer





Date of NHS Lothian

decision

plerixafor 20mg/mL solution for injection

(Mozobil®)

Sanofi Aventis

10.02.20



plerixafor (Mozobil®) is accepted for use within NHSScotland.

Indication under review: in combination with granulocyte-colony stimulating factor (G-CSF) to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and

subsequent autologous transplantation in children aged 1 year to <18 years with lymp h o ma o r solid malignant tumours, either:

- pre-emptively, when circulating stem cell count on the predicted day of collection after adequate mobilisation with G-CSF (with or without chemotherapy) is expected to be insufficie n t

with regards to desired hematopoietic stem cells yield, or - who previously failed to collect sufficient haematopoietic stem cells.

SMC has previously accepted plerixafor for use in adults, in combination with G-CSF to enhance

mobilisation of haematopoietic stem cells to the peripheral blood for collection and subse qu e nt autologous transplantation in patients with lymphoma and multiple myeloma whose cells

mobilise poorly (SMC No. 594/09).



March 2020

polatuzumab vedotin 140mg powder for concentrate for solution for infusion

(Polivy®)

Roche Products Ltd

07.09.20 SMC Report No. 2282


polatuzumab vedotin (Polivy®) is accepted for use within NHSScotland on an interim basis

subject to ongoing evaluation and future reassessment.

Indication under review: in combination with bendamustine and rituximab for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma who are not candidates

for haematopoietic stem cell transplant. In a phase Ib/II study polatuzumabvedotin in combination with bendamustine and rituximab

significantly increased complete response rate compared to bendamustine and rituximab alone. This advice applies only in the context of an approved NHSScotland Patient Acce ss Sch eme


Formulary classification not yet decided – waiting for information from clinician.

pomalidomide 1mg, 2mg, 3mg and 4mg

hard capsules (Imnovid®)

Celgene Ltd

08.12.14



pomalidomide (Imnovid®) is accepted for use within NHS Scotland in combination with

dexamethasone for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior treatment regimens, including lenalidomid e a n d

bortezomib, and have demonstrated disease progression on the last therapy. Pomalidomide plus dexamethasone significantly increased progression-free survival comp a re d

with high-dose dexamethasone in patients with refractory or relapsed and refractory multiple myeloma.

This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e cost-effectiveness of pomalidomide. This advice is contingent upon the continuing availability o f

the patient access scheme in NHS Scotland or a list price that is equivalent or lower.



January 2015

pomalidomide 1mg, 2mg, 3mg and 4mg

hard capsules (Imnovid®)

Celgene Ltd

12.08.19


NOT RECOMMENDED: pomalidomide (Imnovid®)is not recommended for use within NHS

Scotland. Indication under review:In combination with bortezomib and dexamethasone for the treatment of

adult patients with multiple myeloma who have received at least one prior treatment regimen including lenalidomide.


NOT RECOMMENDED



09 September 2020


78/97

Product

Manufacturer





Date of NHS Lothian

decision

ponatinib 15mg, 45mg film-coated tablets

(Iclusig®)

ARIAD pharmaceuticals, Inc.

13.04.15


ponatinib (Iclusig®) is accepted for use within NHS Scotland for adult patients with chronic

phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent

treatment with imatinib is not clinically appropriate; or who have the T315I mutation. Or adult patients with Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ALL) who

are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

A non-comparative phase II study of ponatinib was conducted with primary outcomes of major cytogenetic response in patients with baseline chronic phase CML and major haematologic

response in patients with baseline accelerated or blast phase CML or Ph+ALL. Ponatinib demonstrated efficacy in heavily pre-treated CML and Ph+ALL patients who had received

dasatinib/nilotinib as second line or further line tyrosine kinase inhibitor therapy or who had the T315I mutation.




July 2015

posaconazole 100mg gastro-resistant

tablets (Noxafil®)

MSD Limited

13.10.14

SMC Report No.999/14 PRODUCT UPDATE


posaconazole tablets (Noxafil®) is accepted for restricted use within NHS Scotland in the

treatment of the following fungal infections in adults:

• Invasive aspergillosis in patients with disease that is refractory to amphotericin B or

itraconazole or in patients who are intolerant of these medicinal products;

• Fusariosis in patients with disease that is refractory to amphotericin B or in patients who are intolerant of amphotericin B;

• Chromoblastomycosis and mycetoma in patients with disease that is refractory to itraconazole or in patients who are intolerant of itraconazole;

• Coccidioidomycosis in patients with disease that is refractory to amphotericin B,

itraconazole or fluconazole or in patients who are intolerant of these medicinal products. for prophylaxis of invasive fungal infections in the following patients:

• Patients receiving remission-induction chemotherapy for acute myelogenous leukemia (AML) or myelodysplastic syndromes (MDS) expected to result in prolonged neutropenia and who are at high risk of developing invasive fungal infections;

• Hematopoietic stem cell transplant (HSCT) recipients who are undergoing high -dose immunosuppressive therapy for graft versus host disease and who are at high risk of developing invasive fungal infections.

SMC restriction: to patients in whom there is a specific risk of Aspergillus infection or where fluconazole or itraconazole are not tolerated on the advice of local microbiologists or specialists

in infectious diseases. Posaconazole plasma concentrations are generally higher following administration of

posaconazole tablets than posaconazole oral suspension. The tablet and oral susp e nsio n a re therefore not to be used interchangeably. While the tablets are cost saving when admin istere d

for treatment they are significantly more expensive than the oral suspension when administere d for prophylaxis.

Included on the LJF, for Specialist Use

only, for use in immunocompromised patients for prophylaxis of invasive fun g a l

infections.

October 2014



09 September 2020


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Product

Manufacturer





Date of NHS Lothian

decision

rabbit anti-human thymocyte

immunoglobulin, 25mg powder for solution for infusion (Thymoglobuline

®)

Genzyme Therapeutics Ltd

11.08.08 SMC Report No. 489/08

NOT RECOMMENDED:rabbit anti-human thymocyte immunoglobulin, 25mg powder forsolutio n

for infusion (Thymoglobuline®) is not recommended for use within NHS Scotland for preve n t io n

of graft rejection in renal transplantation. Compared with an alternative agent for induction of immunosuppression it was associated with a lower rate of acute rejection but this did not translate into improved patient or graft survival within

the 12-month study period. The manufacturer has not presented a sufficiently robust econo mic analysis to gain acceptance by SMC.

Rabbit anti-human thymocyte immunoglobulin is also licensed for the treatment of steroid resistant graft rejection in renal transplantation and for the prevention of graft rejection in h e a rt

transplantation. The manufacturer’s submission related only to the prevention of graft reject io n in renal transplantation. SMC cannot recommend the use of rabbit anti-human thymocyte

immunoglobulin for these additional indications.

NOT RECOMMENDED

radium-223 dichloride 1000kBq/mL solution for injection (Xofigo

®)

Bayer Pharma AG

12.10.15 SMC Report No. 1077/15


radium-223 dichloride (Xofigo®) is accepted for use within NHS Scotland.

Indication under review: for the treatment of adults with castration-resistant prostate cancer,

symptomatic bone metastases and no known visceral metastases. In a randomised phase III study of adult men with castration-resistant prostate cancer with

symptomatic bone metastases and no known visceral metastases, treatment with ra d iu m -2 2 3 dichloride was associated with a significant improvement in overall survival compared to

placebo. This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e

cost-effectiveness of radium-223 dichloride. This advice is contingent upon the continuing availability of the patient access scheme in NHS Scotland or a list price that is equivalent or

lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.


question.

December 2015

ramucirumab (Cyrmaza®) 10 mg/mL



09.05.16 SMC Report No. 1156/16

NON SUBMISSION

NOT RECOMMENDED: ramucirumab (Cyramza®) is not recommended for use within NHS

Scotland.

Indication under review: in combination with FOLFIRI (irinotecan, folinic acid, and 5-fluorouracil) for the treatment of adult patients with metastatic colorectal cancer with disease progression o n

or after prior therapy with bevacizumab, oxaliplatin and a fluoropyrimidine. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

ramucirumab (Cyrmaza®) 10 mg/mL


13.06.16



Scotland in combination with docetaxel for the treatment of adult patients with locally adva n ce d or metastatic non-small cell lung cancer with disease progression after platinum-based

chemotherapy. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED



09 September 2020


80/97

Product

Manufacturer





Date of NHS Lothian

decision

ramucirumab (Cyramza®) 10 mg/mL


11.07.16



Scotland. Indications under review:

• In combination with paclitaxel for the treatment of adult patients with advanced gastric cancer or gastro-oesophageal junction adenocarcinoma with disease progre ssio n a f ter prior platinum and fluoropyrimidine chemotherapy

• As monotherapy for the treatment of adult patients with advanced gastric cancer or gastro-oesophageal junction adenocarcinoma with disease progression after prior

platinum or fluoropyrimidine chemotherapy, for whom treatment in combination with paclitaxel is not appropriate

The holder of the marketing authorisation has not made a submission to SMC regarding this product in these indications. As a result we cannot recommend its use within NHS Scotland.

NOT RECOMMENDED

ramucirumab (Cyramza®) 10 mg/mL


11.11.19


NOT RECOMMENDED: ramucirumab (Cyramza®) is not recommended for use within

NHSScotland. Indication under review: As monotherapy for the treatment of adult patients with a d va nce d o r

unresectable hepatocellular carcinoma who have a serum alpha fetoprotein of ≥ 400 ng/mL an d who have been previously treated with sorafenib.


NOT RECOMMENDED

recombinant E.coli asparaginase 10,000

units powder for concentrate for solution for infusion (Spectrila

®)

medac Pharma LLP

09.04.18 SMC Report No. 1319/18


asparaginase (Spectrila®) is accepted for use within NHS Scotland.

Indication under review: as a component of antineoplastic combination therapy for the treatment of acute lymphoblastic leukaemia (ALL) in paediatric patients from birth to 18 years and adults.

Asparaginase produced in E. coli cells has been used in NHS Scotland as an unlicensed medicine as part of treatment of ALL in children and adults; asparaginase (Spectrila

®) provides a

licensed alternative.



April 2018

regorafenib 40mg film-coated tablet

(Stivarga®)

Bayer plc

13.04.15


regorafenib (Stivarga®) is accepted for use within NHS Scotland as treatment of adult p a t ien ts

with unresectable or metastatic gastrointestinal stromal tumours (GIST) who progresse d o n o r are intolerant to prior treatment with imatinib and sunitinib.

In a study of patients with metastatic or unresectable GIST who had prior treatment with imatinib and sunitinib, treatment with regorafenib prolonged the median progression free survival b y 3 .9

months when compared with placebo. This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e

cost-effectiveness of regorafenib and is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.


Includedon the Additional List, Specialist


May 2015

regorafenib (Stivarga®) 40mg film-coated

tablets

Bayer Plc

09.11.15 SMC Report No. 1118/15

NON SUBMISSION

NOT RECOMMENDED: regorafenib (Stivarga®) is not recommended for use within NHS

Scotland.

Indication under review:Adult patients with metastatic colorectal cancer (CRC) who h a ve b e e n previously treated with, or are not considered candidates for, available therapies


NOT RECOMMENDED



09 September 2020


81/97

Product

Manufacturer





Date of NHS Lothian

decision

regorafenib (Stivarga®) 40mg film-coated

tablets Bayer plc

07.05.18


Following a full submission assessed under the end of life process, regorafenib (Stivarga®) is

accepted for use within NHS Scotland. Indication under review: as monotherapy for the treatment of adult patients with hepato cellu la r

carcinoma who have been previously treated with sorafenib. In a randomised, double-blind, phase III study in patients with hepatocellular ca n ce r th a t h ad

progressed on sorafenib treatment, regorafenib significantly improved overall survival compared with placebo on a background of best supportive care.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of regorafenib. This advice is contingent upon the continuing availability of




August 2018

ribociclib 200mg film-coated tablets

(Kisqali®)


12.03.18


ribociclib (Kisqali®) is accepted for use within NHS Scotland.

Indication under review: In combination with an aromatase inhibitor, for the treatment of postmenopausal women with hormone receptor (HR)-positive, human epidermal growth fa cto r

receptor 2 (HER2)-negative locally advanced or metastatic breast cancer as initial endocrine-based therapy.

A phase III double-blind, randomised controlled study demonstrated that ribociclib plus an aromatase inhibitor significantly improved progression-free survival compared with aromatase

inhibitor monotherapy in postmenopausal women with HR-positive, HER2-negative locally advanced or metastatic breast cancer who had not previously received systemic therapy for

advanced disease. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of ribociclib. This advice is contingent upon the continuing availa bil it y o f the PAS in NHS Scotland or a list price that is equivalent or lower.




July 2018

ribociclib 200mg film-coated tablets (Kisqali

®)


11.11.19 SMC Report No. 2198


ribociclib (Kisqali®) is accepted for restricted use within NHSScotland.

Indication under review: for the treatment of women with hormone receptor (HR)-positive,

human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer in combination with fulvestrant* as initial endocrine-based therapy, o r in wo me n

who have received prior endocrine therapy. SMC restriction: women who have relapsed on or within 12 months of completing (neo) adjuvant

endocrine therapy, or those who have progressed on first-line endocrine-based therapy for advanced breast cancer.

Ribociclib in combination with fulvestrant significantly increased progression-free survival

compared with endocrine monotherapy in women with HR-positive, HER2-negative locally advanced or metastatic breast cancer.

This SMC advice takes account of the benefit of Patient Access Schemes (PAS) th a t imp ro ve the cost effectiveness of ribociclib and fulvestrant. This advice is contingent upon the continuing

availability of these PAS in NHSScotland or list prices that are equivalent or lower.


* For SMC advice relating to the use of ribociclib in combination with an aromatase in h ib ito r in this setting, please refer to SMC 1295/18



December 2019



09 September 2020


82/97

Product

Manufacturer





Date of NHS Lothian

decision

rituximab (MabThera) Roche

07.03.03


rituximab(MabThera) is recommended for use by oncologists or haematologists in Scotland who have expertise in treating lymphoma. It should be administered in a hospital enviro n men t

where full resuscitation facilities are available.


note, for Specialist Use only.

September 2004

rituximab (MabThera®)

Roche

13.12.04 SMC Report No. 135/04

rituximab(MabThera®) is accepted for use within NHS Scotland for the treatment of p re vio u sly

untreated patients with stage III-IV follicular lymphoma in combination with cyclophosp ha mid e , vincristine and prednisolone (CVP) chemotherapy.

Rituximab is for use only by oncologists or haematologists who have expertise in treating lymphoma. It should be administered in a hospital environment where full resuscitation facilities

are available. Limited results show that rituximab plus CVP significantly increased th e t ime to treatment failure compared with CVP alone.


note, for Specialist Use only.

March 2005

rituximab 10mg/mL concentrate for

infusion (MabThera®)

Roche

11.12.06


rituximab (MabThera®) is accepted for restricted use within NHS Scotland as maintenance

therapy for patients with relapsed/refractory follicular lymphoma responding to induction therapy with chemotherapy with or without rituximab.

In a phase lll, randomised, open-label study, rituximab maintenance treatment significantly increased the median progression-free survival from 15 months in the observation arm to 52

months in the rituximab arm with an increase in overall survival at three years. This pro lo n g e d survival requires to be confirmed in longer term follow up.

Rituximab is restricted for use only by oncologists or haematologists who have expertise in treating lymphoma.


Use only.

May 2007

rituximab, 100mg and 500mg concentrate

for solution for infusion (MabThera®)

Roche

January 2012


NICE MTA 243 Rituximab for the first-line treatment of stage III–IV follicular lymphoma (review

of NICE technology appraisal guidance 110) states that rituximab, in combination with:

• cyclophosphamide, vincristine and prednisolone (CVP)

• cyclophosphamide, doxorubicin, vincristine and prednisolone (CHOP)

• mitoxantrone, chlorambucil and prednisolone (MCP)

• cyclophosphamide, doxorubicin, etoposide, prednisolone and interferon-α (CHVPi), or chlorambucil

is recommended as an option for the treatment of symptomatic stage III and IV follicular lymphoma in previously untreated people.


Use only.

March 2011



Roche

08.06.09


rituximab (MabThera®) is accepted for restricted use within NHS Scotland for first-line treatme n t

of patients with chronic lymphocytic leukaemia (CLL) in combination with fludarabine and cyclophosphamide.

Rituximab in combination with fludarabine and cyclophosphamide resulted in significantly longer progression free survival than fludarabine and cyclophosphamide alone. The patient populat io n

in the pivotal clinical study had an Eastern Cooperative Oncology Group Performance Status o f 0 or 1 and was a younger population than that generally seen in practice. Evidence in pa t ie n ts

over 70 years of age is limited. Rituximab is restricted to use by specialists in haematology and haemato-oncology.


Use only.

December 2009



Roche

18.01.10


rituximab (MabThera®) is accepted for restricted use within NHS Scotland.

Licensed indication under review: for the treatment of patients with previously u n t re ate d a n d relapsed/refractory chronic lymphocytic leukaemia (CLL) in combination with chemotherapy.

Rituximab in combination with fludarabine and cyclophosphamide resulted in significantly longer progression-free survival than fludarabine and cyclophosphamide alone. The patient population

in the pivotal clinical study had an Eastern Cooperative Oncology Group Performance Status o f 0 or 1 and was a younger population than that generally seen in clinical practice. Evid e n ce in

patients over 70 years of age is limited. Restriction: Rituximab is restricted to use by specialists in haematology and haemato-oncology.

Added to the Additional List, Specialist

Use only.

April 2011



09 September 2020


83/97

Product

Manufacturer





Date of NHS Lothian

decision

rituximab, 100mg in 10mL, 500mg in

50mL, concentrate for solution for infusion (MabThera

®)


07.02.11 SMC Report No. 675/11

rituximab (MabThera®) is accepted for restricted use within NHS Scotland.

Indication under review: Rituximab maintenance therapy is indicated for the treatment of follicular lymphoma patients responding to induction therapy.

SMC restriction: for maintenance treatment in follicular lymphoma patients who have responded to induction with rituximab plus chemotherapy.

Rituximab significantly increased progression free survival following a response to induction therapy in patients with previously untreated follicular lymphoma compared with observation

alone. Longer follow up is required to establish benefit in overall survival.


Use only.

March 2011

rituximab (Mabthera®)

Roche


In combination with bendamustine for first-line treatment of chronic lymphocytic leukaemia. Added to the Additional List, for Specialist Use only.

April 2014

rituximab 1400mg solution for

subcutaneous injection (Mabthera®)


07.07.14


rituximab subcutaneous injection (Mabthera®) is accepted for restricted use within NHS Scotland

for non-Hodgkin’s lymphoma (NHL) in adults: - previously untreated patients with stage III-IV follicular lymphoma in combination with

chemotherapy; - maintenance therapy is indicated for the treatment of follicular lymphoma patients respo nd ing

to induction therapy; - treatment of patients with CD20 positive diffuse large B cell - non-Hodgkin's lymphoma in

combination with CHOP (cyclophosphamide, doxorubicin, vincristine, prednisolone) chemotherapy.

SMC restriction: Subcutaneous rituximab is accepted for use in line with previous SMC a d vice for intravenous rituximab i.e. accepted within licensed indication as above except in the

maintenance setting, where use is restricted to patients who have responded to induction therapy with rituximab plus chemotherapy.

In two pharmacokinetic-based clinical bridging studies, rituximab subcutaneous injection was shown to be non inferior to rituximab intravenous infusion for trough concentration and area

under the concentration time curve. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of rituximab subcutaneous injection. This SMC advice is contingent upon the continuing availability of the patient access scheme in NHS Scotland or a list price that is

equivalent or lower.



October 2014

rituximab with dose adjusted EPOCH

(R-DA-EPOCH)


Treatment of primary mediastinal B-cell lymphoma.


regional guidance.Included on the Additional List, for Specialist Use only.

Categorised RED under the ADTC ‘Policy


November 2018

rituximab, methotrexate, cytarabine and

thiotepa (MATRIX)


Treatment of central nervous system lymphoma. Routinely available in line with local or




April 2017



09 September 2020


84/97

Product

Manufacturer





Date of NHS Lothian

decision

rolapitant (Varuby®) 90mg tablets

Tesaro UK Ltd

04.08.17 SMC Report No. 1266/17


rolapitant (Varuby®) is accepted for restricted use within NHS Scotland.

Indication under review: Prevention of delayed nausea and vomiting associated with highly a n d moderately emetogenic cancer chemotherapy in adults. Rolapitant is given as part of

combination therapy. SMC restriction: as a first-line option in adults undergoing highly emetogenic chemotherapy

(HEC). In phase III studies of patients scheduled to receive highly or moderately emetogenic

chemotherapy,a greater proportion of patients treated with rolapitant-based combination therapy achieved acomplete response (defined as no emesis or use of rescue medication) in the

delayed phase (>24to 120 hours after initiation of chemotherapy) of cycle one co mp a re d with combination therapy alone.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves thecost-effectiveness of rolapitant. This advice is contingent upon the continuing availa b il it y o f

the PASin NHS Scotland or a list price that is equivalent or lower.


guidance. Included on the Additional L ist , for Specialist Use only

July 2018

rucaparib 200mg, 250mg and 300mg film-coated tablets (Rubraca

®)

Clovis Oncology UK Ltd

12.08.19 SMC Report No. 2221

NON SUBMISSION

NOT RECOMMENDED: rucaparib (Rubraca®)is not recommended for use within NHS Scotland.

Indication under review: as monotherapy treatment of adult patients with platinum sensitive,

relapsed or progressive, BRCA mutated (germline and/or somatic), high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have been treated with two or more prior l in e s

of platinum based chemotherapy, and who are unable to tolerate further platinum based chemotherapy.

The holder of the marketing authorisation has not made a submission to SMC regard ing this product in this indication. As a result we cannot recommend its use within NHS Scotland.

NOT RECOMMENDED

rucaparib 200mg, 250mg, 300mg

film-coated tablets (Rubraca®)

Clovis Oncology UK Ltd

09.03.20 SMC Report No. 2224



rucaparib (Rubraca®) is accepted for restricted use within NHSScotland.

Indication under review: As monotherapy for the maintenance treatment of adult p a t ien ts with

platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube, or primary p e rito n e a l cancer who are in response (complete or partial) to platinum-based chemotherapy.

SMC restriction: to patients who do not have a BRCA mutation Rucaparib significantly improved progression free survival compared with placebo in a phase I I I

study in patients with platinum-sensitive serous or endometrioid ovarian, primary perito n e a l o r fallopian tube carcinoma who had received at least two previous platinum based chemothera py

regimens. This advice applies only in the context of an approved NHSScotland Patient Acce ss Sch eme

(PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/list price that is equivalent or lower.




August 2020



09 September 2020


85/97

Product

Manufacturer





Date of NHS Lothian

decision

ruxolitinib (Jakavi®) 5mg, 15mg and 20mg

Tablets Novartis Pharmaceuticals UK Ltd

09.03.15


ruxolitinib (Jakavi®) 5mg, 15mg and 20mg Tablets is accepted for use within NHS Scot la n d a s

thetreatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera

myelofibrosis or post essential thrombocythaemia myelofibrosis. In patients with myelofibrosis, a significantly greater proportion of patients achieved a spleen

response (reduction in spleen volume of at least 35% from baseline) at 48 weeks when tre a ted with ruxolitinib compared with best available therapy. Ruxolitinib was also associated with a

greater proportion of patients reporting a clinically significant reduction in myelofibrosis-re la te d symptoms when compared with placebo.

This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e cost effectiveness of ruxolitinib. It is contingent upon the continuing availabilit y o f th e Pa t ie n t

Access Scheme in NHS Scotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.



April 2015

ruxolitinib phosphate (Jakavi®) 5mg,

10mg, 15mg and 20mg tablets


09.12.19 SMC Report No. 2213


ruxolitinib phosphate (Jakavi®) is accepted for use within NHSScotland.

Indication under review: The treatment of adult patients with polycythaemia vera who are

resistant to or intolerant of hydroxyurea (hydroxycarbamide). Ruxolitinib was superior to best available therapy in two phase III studies in patients with

polycythaemia vera who were resistant to or intolerant of hydroxycarbamide, with or without splenomegaly.


or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.



July 2020

sorafenib 200mg tablets (Nexavar®)

Bayer Plc

13.11.06 SMC Report No. 321/06

NOT RECOMMENDED: sorafenib (Nexavar®) is not recommended for use within NHS Scotlan d

for the treatment of patients with advanced renal cell carcinoma who have failed prior interferon-alfa or interleukin-2 based therapy or are considered unsuitable for such therapy.

Sorafenib has been compared with best supportive care and has been shown to increase progression-free survival, though the impact on overall survival is uncertain. The cost

effectiveness of sorafenib has not been demonstrated.

NOT RECOMMENDED

sorafenib 200mg film-coated tablets

(Nexavar®)

Bayer Plc.

13.07.15


sorafenib (Nexavar®)is accepted for use within NHS Scotland.

Indication under review: treatment of patients with progressive, locally advanced or metasta t ic, differentiated thyroid carcinoma, refractory to radioactive iodine.

Treatment with sorafenib demonstrated a significant, clinically relevant five-month improveme n t in median progression free survival compared with placebo in patients with progressive, lo ca lly

advanced or metastatic, differentiated thyroid carcinoma, refractory to radioact ive iodine. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of sorafenib. This advice is contingent upon the continuing availabil it y o f the PAS in NHS Scotland or a list price that is equivalent or lower.




September 2015



09 September 2020


86/97

Product

Manufacturer





Date of NHS Lothian

decision

sorafenib 200mg film-coated tablets

(Nexavar®)

Bayer plc

11.01.16


nd RESUBMISSION


sorafenib (Nexavar®) is accepted for restricted use within NHS Scotland.

Indication under review: the treatment of hepatocellular carcinoma. SMC restriction: in patients with advanced hepatocellular carcinoma who have failed or are

unsuitable for surgical or loco-regional therapies. In a phase III study in patients with advanced hepatocellular carcinoma, sorafenib was supe rio r

to placebo in terms of overall survival, but not for time to symptomatic progression. This advice takes account of the benefits of a Patient Access Scheme (PAS) that improve s th e

cost-effectiveness of sorafenib. This advice is contingent upon the continuing availability o f th e PAS in NHS Scotland or a list price that is equivalent or lower.




April 2016

sunitinib 12.5mg, 25mg, 50mg capsules

(Sutent®)

Pfizer Ltd

March 2009


sunitinib (Sutent®) is recommended as a first-line treatment option for people with advanced

and/or metastatic renal cell carcinoma who are suitable for immunotherapy and have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.


Use only.

July 2010

sunitinib 50mg capsule (Sutent®)

Pfizer

09.11.09 SMC Report No. 275/06


sunitinib (Sutent®) is accepted for use within NHS Scotland for the treatment of unresectable

and/or metastatic malignant gastrointestinal stromal tumour (GIST) after failure of imatinib mesilate treatment due to resistance or intolerance.

Sunitinib compared with placebo delayed tumour progression by approximately five months. Treatment with sunitinib should not be continued if there is evidence of unacceptable toxicity

or progression of disease. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of sunitinib. This SMC advice is contingent upon the continuing availability of the patient access scheme in NHS Scotland.


Use only.

July 2010

sunitinib 50mg capsule (Sutent®)

Pfizer

12.02.07


NOT RECOMMENDED: sunitinib (Sutent®) is not recommended for use within NHS Scotland for

the treatment of advanced and/or metastatic renal cell carcinoma after failure of interferon -alpha

or interleukin-2 therapy. In uncontrolled trials, sunitinib has been associated with tumour responses in patients who have

metastatic renal cell cancer. However, the economic case has not been demonstrated.

NOT RECOMMENDED

sunitinib 12.5mg, 25mg, 37.5mg, 50mg hard capsules (Sutent

®)

Pfizer Limited

July 2017 NICE MTA 449 supersedes SMC Report

No. 698/11 Patient Access Scheme

NICE MTA 449 Everolimus and sunitinib for treating unresectable or metastatic neuroendocrine tumours in people with progressive disease

Everolimus and sunitinib are recommended, within their marketing authorisations, as options for treating well- or moderately-differentiated unresectable or metastatic neuroendocrine tumours

(NETs) of pancreatic origin in adults with progressive disease.

Added to the Additional List, for Specialist use only.

August 2017

tacrolimus, 5mg/mL concentrate for

infusion and 0.5mg, 1mg, 5mg hard capsules (Prograf

®)

Astellas Pharma Ltd

12.02.07 SMC Report No. 346/07

tacrolimus (Prograf®) is accepted for restricted use within NHS Scotland for the prophylaxis of

transplant rejection in heart allograft recipients. It has shown comparable efficacy to ciclosporin-based regimens in prevention of acute rejection.

It is restricted to use in patients where ciclosporin is not suitable.

Added to the Additional List, if initiate d b y

specialists working in Heart Transplantation.

Shared care for use in kidney and liver

transplants.

December 2008



09 September 2020


87/97

Product

Manufacturer





Date of NHS Lothian

decision

tacrolimus 0.5mg, 1mg, 5mg prolonged-

release capsule (Advagraf®)

Astellas Pharma

10.09.07



tacrolimus (Advagraf®) is accepted for use within NHS Scotland for prophylaxis of transplant

rejection in adult kidney or liver allograft recipients and treatment of allograft rejection re sista n t to treatment with other immunosuppressive medicinal products in adult patients.

It is suitable for use by patients for whom tacrolimus is an appropriate choice of immunosuppressive therapy. It has similar costs per equivalent dose to the tacrolimus

immediate release capsule.

New formulation of a drug already included

in the Formulary.

October 2007

tacrolimus granules for oral suspension (Modigraf

®)

Astellas Pharma Ltd

13.12.10 SMC Report No. 657/10


tacrolimus granules for Oral Suspension (Modigraf®) are accepted for restricted use within NHS

Scotland.

Indication under review: • Prophylaxis of transplant rejection in adult and paediatric, kidney, liver or heart allograft

recipients. • Treatment of allograft rejection resistant to treatment with other immunosuppressive medicinal

products in adult and paediatric patients. SMC restriction: for use in patients for whom tacrolimus is an appropriate choice of

immunosuppressive therapy and where small changes (less than 0.5mg) in dosing incre me n ts are required (e.g. in paediatric patients) or seriously ill patients who are unable to swallow

tacrolimus capsules. Modigraf

® granules for oral suspension offer 18% greater bioavailability than immediate relea se

capsules and may have different bioavailability compared to other unlicensed tacrolimus suspensions in use in the past. Careful monitoring and possible dosage chang es a re n e e d ed

when introducing treatment with Modigraf®.

Tacrolimus granules for oral suspension are significantly more expensive than the capsule

formulation.

Added to the Additional List, for use on the advice of a transplant specialist.

March 2011

tacrolimus (as monohydrate) 0.75mg, 1mg and 4mg prolonged- release tablets

(Envarsus®)

Chiesi Ltd

13.04.15

SMC Report No.1041/15 PRODUCT UPDATE


tacrolimus (Envarsus®) prolonged release-tablets are accepted for use within NHS Scotla n d a s

prophylaxis of transplant rejection in adult kidney or liver allograft recipients and treatment of

allograft rejection resistant to treatment with other immunosuppressive medicinal products in adult patients.

Tacrolimus (Envarsus®) is suitable for use by patients for whom tacrolimus is an appropriate

choice of immunosuppressive therapy. It has increased bioavailability compared with other

tacrolimus preparations. Tacrolimus (Envarsus®) has demonstrated non-inferiority to a

tacrolimus immediate-release capsule and has a similar cost per equivalent dose.

Not included on the LJF because clinicians do not support the formulary inclusion.

April 2015

tacrolimus (Adoport®)


Immunosuppression - for new patients who require immediate release tacrolimus.


regional guidance. Included on the LJF a s a first choice, for Specialist Initiation.

April 2018

tegafur/gimeracil/oteracil 15mg/4.35mg/ 11.8mg and 20mg/5.8mg/15.8mg hard

capsules (Teysuno®)

Nordic Pharma Ltd.

10.09.12


tegafur/gimeracil/oteracil (Teysuno®) is accepted for restricted use within NHS Scotla n d a n d is

indicated in adults for the treatment of advanced gastric cancer when given in combination with

cisplatin. SMC restriction: tegafur/gimeracil/oteracil is restricted to use in patients with advance d g a st ric

cancer who are unsuitable for an anthracycline, fluorouracil and platinum triplet first -line regimen.

In a multicentre, randomised, open-label clinical study in adult patients with ad va nce d g a st ric cancer, tegafur/gimeracil/oteracil in combination with cisplatin was non-inferior to an intravenous

fluoropyrimidine plus cisplatin with respect to overall survival.

Not included on the LJF because clinicians do not support the formulary inclusion.

October 2012



09 September 2020


88/97

Product

Manufacturer





Date of NHS Lothian

decision

telotristat ethyl 250mg film-coated tablets

(Xermelo®)

Ipsen Limited

04.05.18 SMC Report No. 1327/18


telotristat ethyl (Xermelo®) is accepted for restricted use within NHS Scotland.

Indication under review: Treatment of carcinoid syndrome diarrhoea in combination with somatostatin analogue therapy in adults inadequately controlled by somatostatin analogue

therapy. SMC restriction: patients with CS diarrhoea who experience an average of four or m o re b o we l

motions per day, despite receiving somatostatin analogue therapy. A phase III double-blind randomised study showed that telotristat ethyl produced a stat ist ica lly

significant greater reduction in the number of daily bowel motions in patients with carcinoid syndrome on stable dose somatostatin analogue therapy compared with placebo.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of telotristat ethyl. This advice is contingent upon the continuing

availability of the PAS in NHS Scotland or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.



August 2018

talimogenelaherparepvec (Imlygic®) 10

6

and 108 plaque forming units (PFU)/mL

solution for injection

Amgen Ltd

08.05.17 SMC Report No. 1248/17

NON SUBMISSION

NOT RECOMMENDED: In the absence of a submission from the holder of the marketing

authorisation, talimogenelaherparepvec (Imlygic®) is not recommended for use within NHS Scotland.

Indication under review: Treatment of adults with unresectable melanoma that is re g io n a lly o r distantly metastatic (Stage IIIB, IIIC and IVM1a) with no bone, brain, lung or other visceral

disease. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

temoporfin (Foscan®)

Biolitec Pharma

10.05.04


NOT RECOMMENDED: temoporfin (Foscan®) is not recommended for use within NHS Scotland

for the palliative treatment of patients with advanced head and neck squamous cell ca rcin o ma

failing prior therapies and unsuitable for radiotherapy, surgery or systemic chemotherapy. It is the first photosensitising drug licensed in the UK for use in photodynamic therapy (PDT) fo r

the treatment of these patients. Its effect in terms of tumour mass reduction and improvement in quality of life were small and were only observed in patients with lesions less than 10mm d e e p ,

which were fully illuminated with activating light. The quality of life benefits resulting from palliation, particularly in this subgroup, were marginal and the economic case for its use over

other palliative treatments was not made.

NOT RECOMMENDED

temozolomide 5, 20, 100 and 250mg

capsules (Temodal®)

Schering Plough UK Ltd

11.12.06


temozolomide (Temodal®) is accepted for restricted use within NHS Scotland for the tre a tme n t

of newly diagnosed glioblastoma multiforme (GBM) concomitantly with radiotherapy and subsequently as monotherapy treatment.

In a three-year follow up of the pivotal phase lll study, a significant survival benefit was seen over placebo in patients with good performance status and favourable prognostic markers.

Temozolamide is restricted to patients who have had a partial or complete macroscopic resection of their tumour and with World Health Organisation (WHO) performance status 0 or 1.

Added to the LJF as first choice ad ju va n t

treatment in patients with grade IV GBM. For Specialist Use only.



April 2007

As per LJF

July 2019

temozolomide 5, 20, 100 and 250mg

capsules


Concurrent with intermediate course radiotherapy and adjuvant for patients with newly

diagnosed glioblastoma.



July 2017

temozolomide 5, 20, 100 and 250mg capsules


Adjuvant treatment following radiotherapy for patients with grade III glioma without 1p19q co-deletion.

Routinely available in line with local or regional guidance. Included on the

Additional List, for Specialist Use only, Categorised RED under the ADTC ‘Po licy


August 2017



09 September 2020


89/97

Product

Manufacturer





Date of NHS Lothian

decision

temsirolimus (Torisel®)

Wyeth Pharmaceuticals

12.04.10 SMC Report No. 617/10

NON SUBMISSION

NOT RECOMMENDED:temsirolimus (Torisel®) is not recommended for use within NHS

Scotland. Licensed indication under review: the treatment of adult patients with relapsed and/or refracto ry

mantle cell lymphoma [MCL]. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

thalidomide, 50mg hard capsule (Thalidomide Pharmion

®)

Celgene Ltd

12.01.09 SMC Report No. 525/08

thalidomide (Thalidomide Pharmion®) is accepted for use within NHS Scotland in co mb in a t io n

with melphalan and prednisone, as first line treatment of patients with untreated multiple

myeloma, aged 65 years or over or ineligible for high dose chemotherapy. Thalidomide is prescribed and dispensed according to the Thalidomide Pharmion Pregnancy

Prevention Programme. In the pivotal trial in patients aged 65 to 75 years, at 51.5 months median follow-up, the additio n

of thalidomide to melphalan and prednisone gave an overall survival advantage of 18.4 months.


July 2009

thiopenta and carmustine


Treatment of central nervous system lymphoma. Routinely available in line with or regio n a l guidance. Included on the Additional L ist ,

for Specialist Use only. Categorised RED under the ADTC ‘Po licy

for the use of unlicensed (and off-label use) Medicines in NHS Lothian.

April 2018

thiotepa 15mg and 100mg powder for concentrate for solution for infusion

(Tepadina®)

AdienneS.r.l.

09.07.12


NOT RECOMMENDED: thiotepa (Tepadina®) is not recommended for use within NHS Scotla n d

in combination with other chemotherapy medicinal products:

1) with or without total body irradiation (TBI), as conditioning treatment prior to allogeneic or autologous haematopoietic progenitor cell transplantation (HPCT) in haematological diseases in

adult and paediatric patients; 2) when high dose chemotherapy with HPCT support is appropriate for the treatment of solid

tumours in adult and paediatric patients. Two uncontrolled, non-randomised studies including patients with advanced non-Hodgkin’s

lymphoma or Hodgkin’s disease have reported data for non -relapse mortality and overall survival.

The submitting company did not present sufficiently robust clinical and economic analyses to gain acceptance by SMC.

NOT RECOMMENDED



09 September 2020


90/97

Product

Manufacturer





Date of NHS Lothian

decision

tisagenlecleucel 1.2 x 106 – 6 x 108 cells

dispersion for infusion (Kymriah®)


11.02.19


tisagenlecleucel (Kymriah®) is accepted for use within NHSScotland.

Indication under review: treatment of paediatric and young adult patients up to 25 years o f a g e with B-cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in

second or later relapse. Tisagenlecleucel was associated with an overall remission rate of 81% within thre e mo n th s o f

treatment in a single-arm, open-label, phase II study in paediatric and young adult patients with CD19+ relapsed or refractory B-cell ALL.

SMC advice takes account of the benefit of Patient Access Schemes (PAS) that improve the cost effectiveness of tisagenlecleucel and is contingent upon the continuing availabil it y o f th is

PAS in NHSScotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.



December 2019

tisagenlecleucel 1.2 x 106 to 6 x 10

8 cells

dispersion for infusion (Kymriah®)

Novartis Pharmaceutical UK Ltd

09.09.19 SMC Report No. 2200


Following a resubmission under the end of life and ultra-orphan medicine process, tisagenlecleucel (Kymriah


Indication under review:for adult patients with relapsed or refractory diffuse large B -cell lymphoma (DLBCL) after two or more lines of systemic therapy.

Tisagenlecleucel was associated with an overall response rate of 53% in a single -arm, open-label, phase II study in patients with relapsed or refractory DLBCL.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of tisagenleceucel. This advice is contingent upon the continuing

availability of the PAS in NHS Scotland or a list price that is equivalent or lower.


for Specialist Use only..

December 2019

tivozanib 890 micrograms and 1,340 micrograms hard capsules, (Fotivda

®)

Eusa Pharma Limited

08.06.18 SMC Report No. 1335/18


tivozanib (Fotivda®) is accepted for restricted use within NHSScotland.

Indication under review: the first-line treatment of adult patients with advanced renal cell

carcinoma and for adult patients who are vascular endothelial growth factor receptor and mammalian target of rapamycin pathway inhibitor-naïve following disease progression after on e

prior treatment with cytokine therapy for advanced renal cell carcinoma (RCC). SMC restriction: to first-line treatment of advanced RCC.

In a phase III, open-label, randomised, controlled study tivozanib increased progression free survival when compared with a multi-kinase inhibitor in patients with advanced RCC.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of tivozanib. This advice is contingent upon the continuing availab il it y o f




January 2019

topotecan 1mg, 4mg powder for concentrate for solution for infusion

(Hycamtin®)

GlaxoSmithKline

07.05.07


NOT RECOMMENDED: topotecan (Hycamtin®) is not recommended for use within NHS

Scotland for the treatment of patients with relapsed small cell lung cancer (SCLC) for wh o m re -

treatment with the first-line regimen is not considered appropriate. In a trial comparing oral topotecan plus active symptom control (ASC) to ASC alone the

difference in median survival was 12 weeks, in favour of the oral topotecan plus ASC group. Topotecan is not available as an oral formulation in the UK, however, in one trial the re sp o n se

rate and median survival duration were similar for oral and IV topotecan groups. The treatment’s cost in relation to its health benefits was not sufficient to gain a cce pta nce b y

SMC.

NOT RECOMMENDED



09 September 2020


91/97

Product

Manufacturer





Date of NHS Lothian

decision

topotecan 1mg, 4mg powder for

concentrate for solution for infusion (Hycamtin

®)

GlaxoSmithKline

10.12.07 SMC Report No. 421/07

topotecan (Hycamtin®) is accepted for restricted use within NHS Scotland in combination with

cisplatin for patients with carcinoma of the cervix recurrent after radiotherapy and fo r p a t ie nts with stage IVB disease. It is restricted to patients who are cisplatin-naïve.

In an open-label study, overall and progression-free survival were significantly longer for cisplatin plus topotecan compared with cisplatin alone. Haematological adverse e ven ts we re

more common in the cisplatin plus topotecan group. The economic submission d e mo n st rate d that topotecan plus cisplatin was cost effective compared to cisplatin alone in cisplatin -naïve

patients. However, the manufacturer’s justification of the treatment’s cost in relation to its health benefit was not sufficient to gain acceptance by SMC for use in patients with previous exposu re

to cisplatin.

Added to the Formulary. November 2008

topotecan (Hycamtin®)

GlaxoSmithKline


Treatment for new patients presenting with metastatic (stage IVb) cervical carcinoma or patients with recurrence of cervical carcinoma outside the radiation field and with cisplatin free interval of

more than 3 months.

Not preferred. November 2008

topotecan 0.25mg, 1mg hard capsules

(Hycamtin®)

GlaxoSmithKline

14.04.09


topotecan capsules (Hycamtin®) are accepted for restricted use within NHS Scotland as

monotherapy for the treatment of adult patients with relapsed small cell lung cancer (SCL C) fo r whom re-treatment with the first-line regimen is not considered appropriate.

The efficacy of topotecan capsules relative to standard IV chemotherapy is unknown. Topotecan capsules are restricted to use in patients for whom standard intravenous chemotherapy is

inappropriate and who would otherwise receive best supportive care. In one study, oral topotecan plus best supportive care (BSC) was superior to BSC alone fo r th e

primary endpoint of median survival.


Use only.

July 2009

topotecan (Hycamtin®)

Merck Pharmaceuticals

April 2016 NICE MTA 389 supersedes previous FC

advice

NOT RECOMMENDED: NICE MTA 389 Topotecan, pegylated liposomal doxorubicin

hydrochloride, paclitaxel, trabectedin and gemcitabine for treating recurrent ovarian cancer Topotecan is not recommended within its marketing authorisation for treating the first recurrence

of platinum-sensitive ovarian cancer. The Appraisal Committee was unable to make recommendations on the use of these technologies for treating platinum-sensitive ovarian

cancer beyond the first recurrence.

NOT RECOMMENDED

trabectedin 0.25mg and 1mg powde r fo r concentrate for solution for infusion

(Yondelis®)

Immedica

09.12.19


NOT RECOMMENDED: trabectedin (Yondelis®) is not recommended for use within NHS

Scotland.

Indication under review: treatment of adult patients with advanced soft tissue sarcoma, after failure of anthracyclines and ifosfamide, or who are unsuited to receive these agents. E f f ica cy

data are based mainly on liposarcoma and leiomyosarcoma patients. Trabectedin, compared with an alkylating chemotherapy, increased progression-free survival but

not overall survival in patients with advanced liposarcoma or leiomyosarcoma who had previously been treated with an anthracycline-based chemotherapy.

The submitting company did not present a sufficiently robust clinical and economic an a lysis to gain acceptance by SMC.


NOT RECOMMENDED

trabectedin (Yondelis®)

Pharma Mar SA Ltd

April 2016


NOT RECOMMENDED:NICE MTA 389 Topotecan, pegylated liposomal doxorubicin hydrochloride, paclitaxel, trabectedin and gemcitabine for treating recurrent ovarian cancer

Trabectedin in combination with PLDH is not recommended within its marketing authorisation for treating the first recurrence of platinum-sensitive ovarian cancer. The Appraisal Committee was

unable to make recommendations on the use of these technologies for treating platinum-sensitive ovarian cancer beyond the first recurrence.

NOT RECOMMENDED



09 September 2020


92/97

Product

Manufacturer





Date of NHS Lothian

decision

trametinib 0.5mg and 2mg film-coated

tablets (Mekinist®)


12.09.16


trametinib (Mekinist®) is accepted for restricted use within NHS Scotland in combination with

dabrafenib for the treatment of adult patients with unresectable or metastatic melanoma with a BRAF V600 mutation.

SMC restriction: to first-line treatment. In two phase III studies, trametinib in combination with dabrafenib improved progression -free

survival and overall survival compared with BRAF inhibitor monotherapy for the first -line treatment of unresectable or metastatic melanoma with BRAF V600 mutation in adults.

This advice takes account of the benefits of Patient Access Schemes (PAS) th a t imp ro ve th e cost-effectiveness of trametinib and dabrafenib. This advice is contingent upon the co n t in uin g

availability of these patient access schemes in NHS Scotland or list prices that are equivalent o r lower.

This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting. Trametinib is also licensed as monotherapy. As the company submission related only to

combination therapy, SMC cannot recommend use as monotherapy.



December 2016

trametinib 0.5mg, 2mg film-coated tablets (Mekinist

®)


10.07.17 SMC Report No. 1264/17

NON SUBMISSION

NOT RECOMMENDED: In combination with dabrafenib for the treatment of adult patie nts with advanced non-small cell lung cancer with a BRAF V600 mutation.

NOT RECOMMENDED

trastuzumab (Herceptin®)


NICE MTA 257 supersedes SMC Report

No. 386/07

NOT RECOMMENDED:NICE MTA 257 states that trastuzumab in combination with an aromatase inhibitor is not recommended for first-line treatment in postmenopausal wome n with

metastatic hormone-receptor-positive breast cancer that overexpresses HER2.

NOT RECOMMENDED

trastuzumab 150mg vial (Herceptin®)

Roche

09.06.06 SMC Report No. 278/06

trastuzumab (Herceptin®) is accepted for restricted use within NHS Scotland for the treatment of

patients with HER2 positive early breast cancer following surgery, chemotherapy (neoadju va nt or adjuvant) and radiotherapy (if applicable).

In the pivotal trial, the addition of one year of 3-weekly trastuzumab after adjuvant chemotherapy significantly increased disease-free survival compared with that in the observation gro u p . Th e

trial excluded patients with a range of cardiovascular conditions and trastuzumab treatme n t fo r early breast cancer is not recommended in such patients. In patients treated with trastu zu ma b

for early breast cancer, monitoring of cardiac function is required before treatment, every th re e months during treatment and for up to two years after treatment has stopped.

Trastuzumab in this indication is restricted to use by breast cancer specialists.

Added to LJF as first choice for use in

patients with HER2 positive early breast cancer following surgery, chemotherapy

and radiotherapy (if applicable). For Specialist Use only.

April 2007

trastuzumab, 150mg powder for concentrate for solution for infusion

(Herceptin®)

Roche Products Ltd.

12.10.15


nd RESUBMISSION

trastuzumab (Herceptin®) is accepted for restricted use within NHS Scotland.

Indication under review: in combination with capecitabine or fluorouracil and cisplatin for the

treatment of patients with HER2 positive metastatic adenocarcinoma of the stomach or g a st ro -oesophageal junction who have not received prior anti-cancer treatment for their metastatic

disease. It is indicated for use only in patients with metastatic gastric cancer whose tumours have HER2 overexpression as defined by IHC2+ and a confirmatory FISH+ result, or IHC 3+, a s

determined by an accurate and validated assay. SMC restriction: for use in patients whose tumours have HER2 overexpression defined by

immunohistochemistry (IHC) 3+ (“HER2 high expresser”). The addition of trastuzumab to doublet chemotherapy improved overall and progression -free

survival and tumour response. This advice takes account of the views from a Patient and Clinician Engagement (PACE)

meeting.


question.

December 2015



09 September 2020


93/97

Product

Manufacturer





Date of NHS Lothian

decision

trastuzumab, 600mg/5mL solution for

injection (Herceptin®)

Roche Products Ltd

13.01.14


trastuzumab (Herceptin®) is accepted for restricted use within NHS Scotland for the treatment of

adult patients with HER2 positive metastatic breast cancer (MBC) and early breast cancer (EBC) in a range of settings.

Trastuzumab should only be used in patients with metastatic or early breast cancer whose tumours have either HER2 overexpression or HER2 gene amplification as determined by an

accurate and validated assay. SMC restriction: Subcutaneous trastuzumab injection is accepted for use in line with p re vio u s

SMC advice for intravenous trastuzumab (this excludes its use in combination with an aromatase inhibitor for the treatment of postmenopausal patients with hormone-receptor positive

MBC, not previously treated with trastuzumab). In a phase III randomised, open-label clinical study in patients with HER2-positive early b re a st

cancer, subcutaneous trastuzumab was non-inferior to intravenous trastuzumab for the co-primary pharmacokinetic and efficacy endpoints of serum trough concentration (C trough) at pre-

dose cycle 8 before surgery and pathological complete response.



April 2014

trastuzumab emtansine, 100mg and 160mg, powder for concentrate for

solution for infusion (Kadcyla®)

Roche Products Ltd.

10.04.17



As a single agent, for the treatment of adult patients with human epidermal growth factor type 2 (HER2)-positive, unresectable locally advanced or metastatic breast cancer who previously

received trastuzumab and a taxane, separately or in combination. Patients should have either:

• Received prior therapy for locally advanced or metastatic disease, or

• Developed disease recurrence during or within six months of completing adjuvant therapy.



May 2017

trastuzumab (Herzuma®)


As a replacement for the existing trastuzumab brand, Herceptin, where IV trastuzumab is indicated and approved.



March 2019

trifluridine/tipiracil (as hydrochloride),

15mg/6.14mg and 20mg/8.19mg film-coated tablets (Lonsurf

®)

Servier Laboratories Limited

13.02.17 SMC Report No. 1221/17


trifluridine/tipiracil (Lonsurf®) is accepted for use within NHS Scotland for the treatment of a d u lt

patients with metastatic colorectal cancer (CRC) who have been previously treated with, o r a re not considered candidates for, available therapies including fluoropyrimidine-, oxalip la tin - a n d

irinotecan-based chemotherapies, anti-vascular endothelial growth factor agents, and anti-epidermal growth factor receptor agents.

Treatment with trifluridine/tipiracil was associated with an improvement in overall surviva l wh e n compared with best supportive care in patients who had received, or were intolerant of, first an d

second-line therapies for metastatic CRC. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of trifluridine/tipiracil. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.




question.

April 2017

triptorelin acetate (Decapeptyl®

SR 11.25mg)

Ipsen Ltd

12.07.04 SMC Report No. 109/04


triptorelin acetate (Decapeptyl® SR 11.25mg) is accepted for use within NHS Scot la n d fo r th e

treatment of advanced prostate cancer in patients for whom the use of triptorelin is appro p ria te

and who would benefit from reduced frequency of administration compared with Decapeptyl®

SR 3mg (every 3 months vs every 28 days).

Added to the Formulary as first choice gonadorelin analogue for advanced

prostate cancer instead of goserelin or leuprorelin.


guidance. Included in the LJF, for Specialist Initiation.

May 2005

As per LJF

July 2019



09 September 2020


94/97

Product

Manufacturer





Date of NHS Lothian

decision

triptorelin 3.75mg (Gonapeptyl Depot®)

Ferring Pharmaceuticals Ltd

08.05.06 SMC Report No. 269/06

NON SUBMISSION

NOT RECOMMENDED:Gonapeptyl Depot® is not recommended for use within NHS Scotland

for the treatment of advanced, hormone-dependent prostate carcinoma. The holder of the marketing authorisation has not made a submission to SMC regarding this


NOT RECOMMENDED

triptorelin (Decapeptyl SR®) 22.5mg

powder and solvent for suspension for

injection Ipsen Ltd

13.06.11



triptorelin pamoate 22.5mg (Decapeptyl SR®) is accepted for use within NHS Scotland.

• Treatment of patients with locally advanced, non-metastatic prostate cancer, as an

alternative to surgical castration.

• Treatment of metastatic prostate cancer. This new preparation of triptorelin allows 6-monthly administration (as triptorelin pamoate in a

22.5mg dose). Triptorelin 11.25mg (as acetate) is administered every 3 months and has previously been accepted by SMC. Bioequivalence of the pamoate and acetate salts ha s b e en

demonstrated and the new preparation is cost neutral. Note: The indication for triptorelin 11.25mg formulation was reworded in 2007 to achieve

consistency Europe-wide and now reads as per the indication for triptorelin 22.5mg.

Added to the Formulary, as a new formulation of a product already included.

May 2011

triptorelin sustained–release 3mg powder for suspension for injection

(Decapeptyl SR®)

Ipsen Ltd

07.10.19


triptorelin (Decapeptyl SR®) is accepted for use within NHSScotland.

Indication under review: As adjuvant treatment in combination with tamoxifen or an a ro ma ta se

inhibitor, of endocrine responsive early stage breast cancer in women at high risk of recurre n ce who are confirmed as premenopausal after completion of chemotherapy.

In premenopausal women with early breast cancer at high risk of recurrence, ovarian suppression (provided by triptorelin, oophorectomy or radiation ablation) plus an aromatase

inhibitor increased disease free survival compared to ovarian suppression plus tamoxifen. In the same patient population, ovarian suppression plus tamoxifen increased disease -free survival

compared with tamoxifen alone. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of triptorelin. This advice is contingent upon the continuing availabil it y o f the PAS in NHSScotland or a list price that is equivalent or lower.



November 2019

vandetanib (Caprelsa®) 100 mg / 300mg

film coated tablets AstraZeneca UK Limited

11.06.12


NOT RECOMMENDED: vandetanib (Caprelsa®) is not recommended for use within NHS

Scotland for the treatment of aggressive and symptomatic medullary thyroid cancer (MTC) in patients with unresectable locally advanced or metastatic disease.


NOT RECOMMENDED

vemurafenib 240mg film-coated tablet (Zelboraf

®)

Roche Products Ltd.

09.12.13 SMC Report No. 792/12


vemurafenib (Zelboraf®) is accepted for restricted use within NHS Scotland as monotherapy fo r

the treatment of adult patients with BRAF V600 mutation-positive unresectable or metastatic

melanoma. SMC restriction: for use in the first-line treatment of BRAF V600 mutation-positive unresectab le

or metastatic melanoma. Vemurafenib significantly increases overall survival and progression-free survival compared with

a current standard chemotherapy for patients with previously untreated unresectable stage I I IC or stage IV melanoma with V600 BRAF mutation.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of vemurafenib. This SMC advice is contingent upon the

continuing availability of the Patient Access Scheme in NHS Scotland or a list price that is equivalent or lower.


question.

February 2014



09 September 2020


95/97

Product

Manufacturer





Date of NHS Lothian

decision

venetoclax 10mg, 50mg and 100mg

film-coated tablets (Venclyxto®)

AbbVie Ltd

07.08.17



venetoclax (Venclyxto®) is accepted for use within NHS Scotland.

Indication under review: As monotherapy for the treatment of chronic lymphocytic leukaemia

(CLL):

• in the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor.

• in the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B-cell receptor pathway inhibitor.

In phase II, non-comparative studies of patients with relapsed / refractory CLL, tre a tme n t with venetoclax was associated with clinically meaningful overall response rates.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of venetoclax. This advice is contingent upon the continuing availability of




April 2018

venetoclax 10mg, 50mg and 100mg

film-coated tablets (Venclyxto®)

AbbVie Ltd

12.08.19


Following a full submission considered under the orphan equivalent process, venetoclax

(Venclyxto®) is accepted for usein combination with rituximab for the treatment of adult patie nts

with chronic lymphocytic leukaemia (CLL) who have received at least one prior therapy.

Progression-free survival was significantly longer in the venetoclax plus rituximab group compared with chemoimmunotherapy in a phase III study of patients with relapsed or refracto ry

CLL. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves

the cost-effectiveness of venetoclax. This advice is contingent upon the continuing availability of the PAS in NHSScotland or a list price that is equivalent or lower.



guidance. Included on the Additional List


July 2020

vinflunine ditartrate 25mg/mL

concentrate for solution for infusion (Javlor

®)

Pierre Fabre Ltd

13.07.15


NOT RECOMMENDED: vinflunine (Javlor®) is not recommended for use within NHS Scotland.

Indication under review: monotherapy for the treatment of adult patients with advanced or metastatic transitional cell carcinoma of the urothelial tract after failure of a prior platinum-

containing regimen. Efficacy and safety of vinflunine have not been studied in patients with performance status ≥ 2.

Vinflunine plus best supportive care was associated with improved survival when compared with best supportive care alone in the second-line treatment of advanced or metastatic tra n sit io na l

cell carcinoma of the urothelial tract in patients with good performance status. The submitting company did not present a sufficiently robust economic analysis and in additio n

their justification of the treatment’s cost in relation to its benefits was not sufficient to gain acceptance by SMC.


NOT RECOMMENDED

vinorelbine 20 and 30mg capsules

(Navelbine® Oral)

Pierre Fabre Ltd

13.06.05


vinorelbine capsule (NavelbineOral) is accepted for restricted use within NHS Scotland for th e

first line treatment of stage lll or lV non-small-cell lung cancer. It is restricted to use by specialist oncologists as an alternative to the intravenous formulation of

vinorelbine. It is more expensive than the intravenous formulation of vinorelbine. However, it s use may allow changes to service delivery that have individual patient or organisational

benefits.

‘Not preferred’ in Lothian.

July 2007



09 September 2020


96/97

Product

Manufacturer





Date of NHS Lothian

decision

vinorelbine 20mg and 30mg capsule

(Navelbine®)

Pierre Fabre Ltd

13.08.07



vinorelbine (Navelbine®) is accepted for restricted use within NHS Scotland for treatment of

advanced breast cancer stage III and IV relapsing after, or refractory to, an anthracycline -containing regimen.

It is restricted to use by specialist oncologists as an alternative to the intravenous formulation o f vinorelbine where vinorelbine is considered to be appropriate. It is more expensive than the

intravenous formulation of vinorelbine. However, its use may allow changes to service d e live ry that have individual patient or organisational benefits.


Use only.

July 2008

vismodegib (Erivedge®) 150 mg hard

capsules

Roche Products Ltd

07.10.13 SMC Report No. 924/13

NON SUBMISSION

NOT RECOMMENDED: vismodegib (Erivdege®) is not recommended for use within NHS

Scotland for the treatment of adult patients with:

• symptomatic metastatic basal cell carcinoma • locally advanced basal cell carcinoma inappropriate for surgery or radiotherapy


NOT RECOMMENDED

Zarzio filgrastim 300mcg and 480mcg pre-filled syringes


For primary or secondary prophylaxis of neutropenia; treatment of profound neutropenia associated with sepsis and mobilisation of autologous stem cells for transplants.

Routinely available in line with local or regional guidance. Included in the LJF, fo r

Specialist Initiation.

May 2017

zoledronic acid (Zometa) Novartis Europharm Ltd

12.05.03 SMC Report No. 29/02

RESUBMISSION

zoledronic acid (Zometa) should be restricted to prescribing by oncologists for patients with breast cancer and multiple myeloma. It provides an alternative to other bisphosphonates licensed for prevention of ske le ta l re la te d

events. It may offer some minor advantages in terms of administration. At a local level the decision will rest on weighing up the additional cost against other options available for improving

the delivery of oncology services. Zoledronic acid has a broader range of indications than other bisphosphonates available

and has shown some efficacy in patients with prostate cancer and NSCLC and other solid tumours. The quality of the economic evidence provided is insufficient to demonstrate that

the use of this product for these indications is cost effective. The licence holder has indicated their decision to appeal.

‘Not Preferred’ in Lothian. A submission has not been made to FC regarding this

product for this indication.

zoledronic acid (Zometa)

Novartis Europharm Ltd

12.01.04 SMC Report No. 29/02

INDEPENDENT REVIEW PANEL ASSESSMENT

NOT RECOMMENDED:zoledronic acid (Zometa) is not recommended for use within NHS

Scotland for the prevention of skeletal related events (SREs) in patients with advanced prostate cancer involving bone.

Although zoledronic acid demonstrated a reduction in SREs compared with placebo in these patients, the absolute reduction was small and the study requires caution in acce p t in g th is a s

sufficient evidence to introduce zoledronic acid into standard practice for the treatment of patients with metastatic prostate cancer. An economic case was submitted by the manufacturer

but its quality was not judged to be sufficient to support a recommendation that the drug is co st effective relative to standard practice in Scotland for this particular indication.

NOT RECOMMENDED

zoledronic acid (Zometa®)

Novartis


For tumour induced hypercalcaemia.

Added to the Formulary as first choice.


regional guidance. Included in the LJF, fo r Specialist Use only.

April 2010



09 September 2020


97/97

Product

Manufacturer





Date of NHS Lothian

decision

zoledronic acid (Zerlinda®) 4mg/100mL

solution for infusion pre-filled bag Actavis


Adjuvant use in women at high risk of recurrence to reduce the rate of

breast cancer recurrence in bone. High risk patients are:

• breast cancer patients (irrespective of whehter they have had chemotherapy)

• who are post-menopausal and deemed by local protocol to be high risk: i.e. ER

low/negative, or ER 4-8 with one or more of the following features – HER2 positive or Grade 3 or

T3/4 or node positive.

• pre-menopausel patients in the same risk category who warrant ovarian suppression.





October 2016

zoledronic acid (Zerlinda®) 4mg/100mL

solution for infusion

Actavis


Prevention of skeletal related events in patients with skeletal metastases from solid tumours excluding breast and prostate cancer.

Routinely available in line with local or regional guidance. Included on the

Additional List, for Specialist Use only.

August 2017



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