horizon scanning why, how and what lies ahead?. key learning points why is horizon scanning for...
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Horizon ScanningHorizon ScanningWhy, how and what lies Why, how and what lies
ahead?ahead?
Key learning points • Why is horizon scanning for medicines important
• How is horizon scanning undertaken
• What information is available to support the managed entry of new drugs and its limitations
• What key therapeutic advances are expected in the next year
What is horizon scanning?Horizon Scanning has been defined as:
‘the systematic examination of potential threats, opportunities and likely future developments…….'
Horizon scanning for medicines aims to identify:• treatments likely to become available to the NHS
that may have significant implications for – clinical practice – service design – finance
• potential disinvestments
Why horizon scan for medicines?
Informs and primes providers and commissioners to proactively implement management strategies
– Anticipate pressures (financial and service delivery)– Manage budgets– Plan services - new and redesign– Identify areas for disinvestment– Manage entry into hospital/ formulary /practice etc– Identify drugs suitable for homecare
A woman with advanced kidney cancer and six months left to live says she is missing out on a potentially life-saving drug…….
Prescribing data (England)
2010: •NHS medicines expenditure £12.9 billion.•Hospital use accounted for 31.7%
NHS Information Centre: Hospital Prescribing England
Drivers of growth in prescribing• New drugs for diseases where previous options were
limited e.g. rare genetic diseases• Expanded indications (increase in eligible population)
e.g. chemotherapy drugs • New drug regimens or maintenance treatments added
to standard therapy e.g. chemotherapy, antidiabetes• Displacement of old drugs with new drugs at higher
cost e.g. “biologicals”, oral anticoagulants• ‘Medicalisation’ e.g. social anxiety• Ageing population
Other factors that impact on management of drug budgets
HTAs (NICE, SMC, AWMSG)
Payment by Results (PbR) tariff
HomecareCancer drug fund
Value based pricing
Commissioning
Innovation Health Wealth
and MHRA consultation
Patient access schemes
Patent expiries
Orphan drugs/ indications
Personalised healthcare
International markets
Patent expiries
• Specialist media for press releases highlighting – conference presentations– dates for submission to
licensing authorities – plans for development
• Specialist websites, databases and journals– UK PharmaScan
• Industry (contacts, websites, annual reports)
• Licensing agencies• Clinical trial registries• Clinical specialists• Other horizon scanners
Information sources used by horizon scanners
UK Horizon scanning organisations
Health & Social Care Northern Ireland
Medicines and Prescribing centre
How does the UKMi horizon scanning process work?
Systematic early identification
(horizon scanning)
PrioritisationInformation
retrieval
Filtration and selection
Assessment Dissemination
UKMi Horizon scanning products
www.nelm.nhs.ukPassword restricted to NHS
Prescribing Outlook
Horizon scanning challenges• Licence extensions/ new formulations are
difficult to track and time frames for approval are shorter
• Company acquisitions vs. in-house R&D (biotech)• Company mergers• Confidentiality issues• Epidemiology or target population may be
difficult to define and quantify
Horizon scanning challenges• Regulatory delays• Differences in views between licensing
authorities• Regulatory transparency differences• Indication applied for may not be the same as
that eventually approved • Cost is rarely known prior to launch• Rate and extent of market uptake is difficult to
forecast
Factors influencing impactDrug specific • Anticipated licence?• Formulation and administration?• First in class?• Place in therapy?• Significant improvement in disease management?• Other trials ongoing? (Licence extensions are easier
to obtain and there may be off label use.)• Cost of drug, administration and testing• What could be its USP (unique selling point)?
Factors influencing impactExternal factors
• Size of target population i.e. large population or significant subset of large population? What is large?
• Will it change where patients are treated e.g. hospital vs. intermediate vs. home vs. primary care?
• Local use (in ongoing clinical trials or unlicensed use)?• Funding of services? E.g National commissioning• Where in NICE agenda?• Which company?• Media/public interest?
Factors UKMi use for prioritisation• significant improvement in disease management?• additional therapy or displacement of existing therapies?• first in class or has a major new indication?• limited other drug/non-drug alternatives?• high cost? • service implications e.g. route/ formulation/ method of
delivery• the drug or disease area is considered an NHS priority• in the EU licensing process• significant additional indications in the advanced pipeline
stage• likely to be significant media interest.
Who is involved in UKMi prioritisation?
• UKMi pharmacists with extensive horizon scanning expertise
• Primary care/ commissioning pharmacists• Secondary care/ interface pharmacists• Other people/organisations with horizon scanning
expertise
• Accessible via NHS Evidence (limited) and UKMi (full access for NHS staff) websites
• Contains over 1300 active monographs• Updated daily• In September 2012– 356 monographs updated– 14 evidence based evaluations added– 2,833 registered users
• Monthly newsletter sent to registered users (NHS only)• Reporting facility (NHS only)
UKMi Horizon scanning productsNew Drugs Online (NDO) database
NDO content• Name (generic, company, synonym)• Indication, formulation• Pharmacology, epidemiology• Key trial data• Stage in licensing process (EU, US), anticipated UK launch
date• Orphan status• Links to independent evaluations e.g. NHSC, LNDG • In NICE pipeline
Other publications relevant to medicines budget planning
• NICE/ SMC/ AWMSG guidance• London New Drugs Group reviews• UKMi Prescribing Outlook cost calculator• UKMi New Medicines Profiles• UKMi IFR summaries• UKMi NICE bites• MPC* Evidence summaries: new medicines• Forward Look (Scotland)• Regional advisory committees e.g. NETAG, MTRAC
* Medicines Prescribing Centre (formerly NPC – National Prescribing Centre)
2012/132012/13Key pressures due to Key pressures due to
new medicines or licence new medicines or licence extensionsextensions
Key new medicines due 2012/13Cardiovascular systemApixaban, rivaroxaban, dabigatran - stroke prevention, VTE treatment and long term prevention
Defibrotide - hepatic veno-occlusive disease
Lomitapide, mipomersen - hypercholesterolaemia
Respiratory Ivacaftor - cystic fibrosis
Aclidinium, glycopyrrolate - COPD
Pirfenidone - pulmonary fibrosis
Neurology/rheumatology Alemtuzumab, laquinimod, dimethyl fumarate, teriflunomide – multiple sclerosisTofacitinib – Rheumatoid arthritisStrontium ranelate - Osteoarthritis
Miscellaneous Insulin degludec - diabetes.
Ruxolitinib - myelofibrosis
Nalmefene - alcohol dependence
Eltrombopag – thrombocytopenia associated with hepatitis C.
Chemotherapy Vismodegib - basal cell carcinoma.
Pertuzumab - breast cancer
Crizotinib - NSCLC
Regorafenib - colorectal cancer
Abiraterone, sipuleucel-T - prostate cancer
Pazopanib, vintafolide – ovarian cancer
Pixantrone - NHL
Key new medicines due 2012/13
UKMi Prescribing Outlook content 2010 Status of featured drugs April 2012
Reasons for delay
• Licensing process raises questions causing – delay– withdrawal from licensing process– discontinuation
• Once licensed the company may not launch in the UK at all
• Waiting for NICE appraisal/ reimbursement negotiations
What happens when managed entry is not planned?
Amifampridine (Firdapse)
• Licensed Feb 2010 for Lambert-Eaton myasthenic syndrome (LEMS) - prevalence of about 1 per 100,000
• Licensed as orphan drug under exceptional circumstances• LEMS previously treated with unlicensed 3,4-
diaminopyridine base; cost about £1,000/year/patient • Amifampridine is phosphate salt formulation of 3,4-
diaminopyridine; cost about £44,000/year/patient• Significant cost and supply pressures resulted
Firdapse ..why missed?• Ultra orphan drug (v. small no. patients in few centres)• LEMS already treated with comparatively cheap
(although unlicensed) drug• Lack of background ‘noise’
– Exceptional circumstances status shortened the licensing process (more difficult to track)
– Evidence required not the same for orphans as for other drugs– Licensing company relatively small and bought in amifampridine
just prior to submitting for a licence– No publicity about filing for a licence
• Price could not be anticipated
Firdapse learning points
• Be aware of currently unlicensed treatments• Be more aware of orphan drugs (database
amended)• EMA filing data now available • Highlights the fact horizon scanning processes
cannot be comprehensive
What can clinical pharmacists do?
• Utilise horizon scanning resources• Be aware of developments within your
speciality• Liaise with key clinicians and budget holders to
raise awareness and ensure new medicines and licence extensions are planned for
What can clinical pharmacists do?
• Understand NHS funding mechanisms and service issues
• Highlight key developments to horizon scanners
Thank youThank youAny questions?