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Submission to Applied Health Economics and Health Policy

Systematic Assessment of Decision-analytic Models for Chronic

Myeloid Leukemia

Ursula Rochau1,2, Ruth Schwarzer1,2, Beate Jahn1,2, Gaby Sroczynski1,2, Martina

Kluibenschaedl1,2, Dominik Wolf3,4, Jerald Radich5, Diana Brixner1,2,6, Guenther

Gastl3, Uwe Siebert1,2,7,8

1 Division of Public Health Decision Modelling, Health Technology Assessment and Health Economics, ONCOTYROL -

Center for Personalized Cancer Medicine, Innsbruck, Austria;

2 Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health

and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall

i.T., Austria;

3 Internal Medicine V, Hematology and Oncology, Medical University Innsbruck, Austria;

4 Internal Medicine III, University of Bonn, Germany;

5 Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA;

6 Department of Pharmacotherapy and Program in Personalized Health Care, University of Utah, Salt Lake City, Utah,

USA;

7 Center for Health Decision Science, Department of Health Policy and Management, Harvard School of Public Health,

Boston, MA, USA;

8 Institute for Technology Assessment and Department of Radiology, Massachusetts General Hospital, Harvard

Medical School, Boston, MA, USA;

1

Corresponding Author:

Dr. Ursula Rochau, MSc

Area 4: Health Technology Assessment and Bioinformatics

ONCOTYROL - Center for Personalized Cancer Medicine

Innrain 66a

A – 6020 Innsbruck

Austria

T: +43(0)50-8648-3947, F: +43(0)50-8648-673947

[email protected]

Online Supplementary Material 1

Literature search

NHS EED via OVID (update search date: 20.8.2013), (Pre-)Medline (update

20.8.2013) via OVID: Search fields: title, text, subject heading word, EMBASE via

Harvard (update 20.8.2013): Search fields: title, abstract, particularity in EMBASE:

adding German syntax

Keywords: (CML OR chronic myelogenous leuk* OR chronic myeloid leuk*)

AND (decision anal* OR decision-anal* OR cost-utility OR cost utility OR cost-benefit

2

OR cost benefit OR cost-minimi* OR cost minimi* OR QALY Or Markov OR cost-

effectiveness OR cost effectiveness OR health care model OR decision model OR

decision-model OR decision tree OR decision-tree OR health care evaluation model

OR discrete event simulation OR discrete OR individual simulation OR transmission

model OR AUC OR area under the curve model OR survival partition model)

MeSH: leukemia, myelogenous, chronic, bcr-abl positive/ or leukemia, myeloid,

chronic, atypical, bcr-abl negative/; markov chain'/ monte carlo method/ 'quality

adjusted life year'/ "Quality of Life"/cost-benefit analysis'/ area under curve'/ cost

utility analysis'/ decision tree'/ Models, Economic/

A restricted search was performed in EconLit via Harvard (update search date:

8.9.2013) and Tufts CEA Registry. Econlit: TI=cml OR TI=(leukemia or leukaemia)

A broader search was performed inTufts CEA Registry.

TI: CML OR Chronic Myeloid Leukemia OR Chronic Myeloid Leukaemia OR chronic

myelogenous leukemia OR chronic myelogenous leukaemia

3

Table II: Summary of Cost-Effectiveness Results and Conclusions of Included Studies[1, 2]

Author, year, country

Study type, currency, index year

Data sources Annual discount rate

Cost-effectiveness/ utility relation in US $ (October 2011) ¥, †

Cost-effectiveness/ utility relation Conclusions

Pre-imatinib era

Beck et al. 2001[3],university medical centers in North America & Europe

CEA, CUA, U.S.$, estimated 2000

Efficacy: RCT (FCMLG)QoL/Utilities: Updated Kattan et al. 1996[4]Costs: Updated Kattan et al. 1996[4]

Benefits and costs: 3%

ICER discountedIFNα vs. HU: $23,491/ LY Cytarabine+IFNα vs. HU: $22,708/ LYCytarabine+IFNα vs. IFNα: $20,881/ LY ICUR discountedIFNα vs HU: $30,930/ QALY Cytarabine+IFNα vs. HU: $27,994/ QALY Cytarabine+IFNα vs. IFNα: $22,056/ QALY

ICER discountedIFNα vs. HU: $18,000/ LY Cytarabine+IFNα vs. HU: $17,400/ LYCytarabine+IFNα vs. IFNα: $16,000/ LY

ICUR discountedIFNα vs. HU: $23,700/ QALY Cytarabine+IFNα vs. HU: $21,450/ QALY Cytarabine+IFNα vs. IFNα: $16,900/ QALY

“For all plausible ranges of the efficacy of IFNα and cytarabine, the combination therapies are cost-effective with respect to chemotherapy alone.” Further,” the increment in costs with cytarabine added to IFNα is more than offset by the extra increment in QALE.”

Kattan et al. 1996[4],university medical centers North America & Europe

CEA, CUA, U.S.$, estimated 1995

Efficacy: RCTs, published studies, clinician panelQoL/Utilities: Clinical panel: Direct scalingCosts: U.S./ 2 European cancer centers, clinical-cost accounting systems


ICER discountedIFNα vs. HU: $39,078/ year of life savedICUR discountedIFNα vs. HU: $51,317/ QALY

ICER discountedIFNα vs. HU: $26,500/ year of life savedICUR discountedIFNα vs. HU: $34,800/ QALY

“Compared with HU, IFNα is, in most clinical scenarios, a cost-effective initial therapy for patients with chronic-phase CML who can tolerate the drug.”

Liberato et al. 1997[5],Italy

CUA, U.S.$, 1995

Efficacy: RCTs, published studies QoL/Utilities: 10 physicians: VAS Costs: Retail drug prices; published literature, expert panel judgments


ICUR discountedIFNα Scenario A vs. chemotherapy: $131,981/ QALY IFNα Scenario B vs. chemotherapy: $93,640/ QALY

ICUR discountedIFNα Scenario A vs. chemotherapy: $89,500/ QALY IFNα Scenario B vs. chemotherapy: $63,500/ QALY

“In conclusion, IFNα adds an effective option to the treatment of CML, but is expensive in the most common protocols.”

4






Messori 1998[6],German/English, Italian/Japanese trials

CEA, U.S.$, estimated 1997

Efficacy: 4 RCTsQoL/Utilities: Not evaluatedCosts: Published literature


ICER discountedIFNα vs. control group 1)German trial: $130,866/ LY 2)English trial: $180,724/ LY 3)Italian trial: $236,617/ LY 4)Japanese trial: $317,214/ LY

ICER discountedIFNα vs. control group 1)German trial: $93,461/ LY 2)English trial: $129,068/ LY 3)Italian trial: $168,985/ LY 4)Japanese trial: $226,545/ LY

“Our cost-effectiveness study gave a 'negative' result because our findings showed that an unselected use of IFNα in CML has an unfavorable pharmacoeconomic ranking.”

Imatinib era

Chen et al. 2009[7],China

CEA, CUA, RMB, estimated 2008

Efficacy: RCTs (incl. IRIS), published studiesQoL/Utilities: Reed et al. 2004[8]Costs: Retail price, unit costs top tier hospitals

Benefits and costs: 3.5%

ICER discounted Imatinib vs. IFNα: $20,463/ LYICUR discounted Imatinib vs. IFNα: $20,126/ QALY

ICER discounted Imatinib vs. IFNα: RMB74,908/ LYICUR discounted Imatinib vs. IFNα: RMB73,674/ QALY

“This study confirms that imatinib is more cost-effective than IFNα from the Chinese public health-care system perspective“.

Dalziel et al. 2004[9],U.K.

CUA, £, 2002

Efficacy: RCTs, published studiesQoL/Utilities: Patients (IRIS): EQ-5D (Imatinib, IFNα), estimates clinical panel from Kattan et al. 1996[4], (HU)Costs: BNF, SUHT, NHS Trust databases

Benefits: 1.5%, Costs: 6%

ICUR discounted (undiscounted) Imatinib vs. IFNα: $52,108/ QALY ($63,217/ QALY)Imatinib vs. HU: $173,033/ QALY ($167,393/ QALY)

ICUR discounted (undiscounted) Imatinib vs. IFNα: £26,180/ QALY (£31,761/ QALY)Imatinib vs. HU: £86,934/ QALY (£84,100/ QALY)

“Imatinib appears to be more effective than current standard drug treatments in terms of cytogenetic response and PFS, with fewer side-effects.”

Gordois et al. 2003[10]U.K.

CUA, £, 2001

Efficacy: RCTs, published studies, clinician panelQoL/Utilities: 6 clinicians using the EQ-5DCosts: Chartered Institute of Public Finance and Accountancy, Dept. of Health, 6 NHS Trusts, published literature


ICUR discountedAccelerated Phase Imatinib vs. comparator: $59,408/ QALY Blast Crisis Phase Imatinib vs. comparator: $85,514/ QALY

ICUR discountedAccelerated Phase Imatinib vs. comparator: £ 29,344/ QALY Blast Crisis Phase Imatinib vs. comparator: £42,239/ QALY

"We conclude that treatment of CML with imatinib confers considerably greater survival and quality of life than conventional treatments but at a cost."

Reed et al. CEA, Efficacy: RCTs (incl. IRIS, Benefit ICER discounted (undiscounted) ICER discounted (undiscounted) “The results of the current study

5






2004[8]U.S.

CUA, U.S.$, 2002

FCMLG), published studiesQoL/Utilities: Patients (IRIS): EQ-5D (imatinib, IFNα+LDAC); no data HU: imatinib utility values used Costs: Medication costs: Red Book, outpatient visits/inpatient costs: Medicare

s and costs: 3%

Imatinib vs. IFNα+LDAC: $53,841/ LYS,($48,844/ LYS)ICUR discounted (undiscounted) Imatinib vs. IFNα+LDAC: $54,091/ QALY ($51,843/ QALY)

Imatinib vs. IFNα+LDAC: $43,100/ LYS,($39,100/ LYS)ICUR discounted (undiscounted) Imatinib vs. IFNα+LDAC: $43,300/ QALY ($41,500/ QALY)

demonstrate that compared with IFNα plus LDAC, imatinib is a cost-effective first-line therapy in patients with newly diagnosed chronic-phase CML.”

Reed et al. 2008[11](update Reed et al. 2004[8]), U.S.

CEA, CUA, U.S.$, 2006

Efficacy: Reed et al. 2004[8]., update IRISQoL/Utilities: Reed et al.2004[8]Costs: Medications: AWP Red Book, WAC, Medi-Span; outpatient visits/ inpatient costs: Medicare


ICER discounted (undiscounted) Imatinib vs. IFNα+LDAC using AWP: $59,679/ LY ($53,657/ LY); using WAC: $48,124/ LY ($43,868/ LY) ICUR discounted (undiscounted) Imatinib vs. IFNα+LDAC using AWP: $63,656/ QALY ($60,050/ QALY); using WAC: $51,370/ QALY ($49,106/ QALY)

ICER discounted (undiscounted) Imatinib vs. IFNα+LDAC using AWP: $53,535/ LY ($48,133/ LY) using WAC: $43,170/ LY ($39,352/ LY) ICUR discounted (undiscounted) Imatinib vs. IFNα+LDAC using AWP: $57,103/ QALY ($53,868/ QALY) using WAC: $46,082/ QALY ($44,051/ QALY)

“Although the analysis revealed that the original survival estimates were conservative, the updated cost-effectiveness ratios were consistent with, or slightly higher than, the original estimates, depending on the method for assigning costs to study medications.”

Warren et al. 2004[12],U.K.

CUA , £, 2001

Efficacy: RCTs, published studiesQoL/Utilities: Panel of clinicians: EQ-5DCosts: BNF, NHS reference costs, Personal Social Services Research Unit, authors' assumptions


ICUR discounted Imatinib vs. HU: $77,880/ QALY

ICUR discounted Imatinib vs. HU: £38,468/ QALY

"In the present model analysis, imatinib as a second-line treatment for patients with chronic phase CML was found to offer considerable health benefits to patients, but at a cost to the payer."

Second-generation TKIsGhatnekar et al.

CEA, CUA, €,

Efficacy: RCT (12 week head-to-head), published literature

Benefits and

ICER discountedDasatinib vs. Imatinib: $8,167/ LY

ICER discountedDasatinib vs. Imatinib: €6,332/ LY

“The results indicate that dasatinib treatment in CML

6






2010[13],Sweden

2008 QoL/Utilities: 100 U.K. lay persons: TTO using the EQ-5DCosts: Treatment practice: 2 Swedish hematologists; Unit cost: FASS, regional tariffs and fees, income distribution survey, consumption and production in Sweden

costs: 3%

ICUR discounted Dasatinib vs. Imatinib: $8,873/ QALY

ICUR discounted Dasatinib vs. Imatinib: €6,880/ QALY

patients resistant to standard dose imatinib in Sweden is a cost-effective treatment in comparison to imatinib 800 mg/daily. Dasatinib is expected to generate greater health benefits at a cost per QALY of about €6,880 with a life-long societal perspective.”

Hoyle et al, 2011[14] (Rogers et al. 2012[15]),U.K.

CEA, CUA, £,2009-2010

Efficacy: several trials from systematic reviewQoL/Utilities: adopted from Reed[8, 11]Costs: Expert opinion, trials, BNF, Curtis

Benefits and costs: 3.5%

Imatinib intolerant:ICER discountedDasatinib vs. IFNα : $76,107/ LY Nilotinib vs. IFNα : $108,833/ LYICUR discountedDasatinib vs. IFNα : $131,132/ QALY Nilotinib vs. IFNα: $166,175/ QALYImatinib resistantICER discountedDasatinib vs. high dose-imatinib: $74,841/ LYNilotinib vs. high dose-imatinib: Nilotinib dominatesICUR discounted Dasatinib vs. high dose-imatinib$145,226/ QALYNilotinib vs. high dose-imatinib: Nilotinib dominates

Imatinib intolerant:ICER discountedDasatinib vs. IFNα: £47,951/ LY Nilotinib vs. IFNα: £68,570/ LYICUR discounted Dasatinib vs. IFNα: £82,619/ QALYNilotinib vs. IFNα: £104,698 QALYImatinib resistant: ICER discountedDasatinib vs. high dose-imatinib: £47,153/ LY Nilotinib vs. high dose-imatinib: Nilotinib dominatesICUR discounted Dasatinib vs. high dose-imatinib: £91,499/ QALYNilotinib vs. high dose-imatinib: Nilotinib dominates

“Whilst clinical data remains immature, the cost-effectiveness of dasatinib and nilotinib for imatinib-resistant people is highly uncertain. Both nilotinib and dasatinib are highly unlikely to be cost-effective versus IFNα for people intolerant to imatinib.”

Loveman et al. 2012[16]

CEA,

CUA , £, 2009-2010

Parameters mostly adopted from Hoyle et al.[14] (Rogers et al.[15]), Drug costs: BNF

Costs and benefits 3.5%

ICUR discounted:Nilotinib vs. HU: $41,956/ QALY

Dasatinib vs. Nilotinib: $79,385/ QALY

ICUR discountedNilotinib vs.HU: £26,434 / QALYDasatinib vs. nilotinib: £50,016 / QALYRemaining strategies were dominated

“Nilotinib and dasatinib are slightly more cost-effective than high-dose imatinib because of slightly lower costs and better

7






Remaining strategies were dominated effectiveness than high-dose imatinib.”“It is not possible to derive firm conclusions about the relative cost-effectiveness of the three interventions owing to the great uncertainty around data inputs.”

Pavey et al. 2012[17]

CEA, CUA, £, 2011-2012

Efficacy: several trials from systematic reviewQoL/Utilities: adapted from Reed[8], Lee[18], Dalziel[9]Costs: BNF, MIMS, Oxford

Outcomes 2009 survey, (NSRC), Curtis, trials and manufacturer submissions

Costs and benefits 3.5%

Scenario 1ICER discountedNilotinib vs. imatinib: $17,657/ LYDasatinib vs. imatinib: $301,643/ LYNilotinib vs. dasatinib: $-142,728/ LYICUR discounted:Nilotinib vs. imatinib: $36,786/ QALYDasatinib vs. imatinib: $609,172/ QALYNilotinib vs. dasatinib: Dasatinib dominatedScenario 2ICUR discounted:Nilotinib vs. imatinib: $29,429/ QALYDasatinib vs. imatinib: $376,686/ QALYNilotinib vs. dasatinib: Dasatinib dominatedScenario 3ICER discountedImatinib+2LNilo vs. nilotinib: Nilotinib cost-savingImatinib+2LNilo vs. dasatinib+2LNilo: $295,757/ LY

Scenario 1ICER discountedNilotinib vs. imatinib: £12,000/ LYDasatinib vs. imatinib: £205,000/ LYDasatinib vs. nilotinib: Dasatinib dominatedICUR discounted:Nilotinib vs. imatinib: £25,000/ QALYDasatinib vs. imatinib: £414,000/ QALYDasatinib vs. nilotinib: Dasatinib dominatedScenario 2ICUR discounted:Nilotinib vs. imatinib: : £20,000/ QALYDasatinib vs. imatinib: £256,000/ QALYDasatinib vs. nilotinib: : Dasatinib dominatedScenario 3ICER discountedImatinib+2LNilo vs. nilotinib: Nilotinib cost-savingImatinib+2LNilo vs. dasatinib+2LNilo: £201,000/ LYNilotinib vs. dasatinib+2LNilo: £356,000/

“… assuming the use ofsecond-line nilotinib, first-line nilotinib appears to be more cost-effective than first-line imatinib for most scenarios. Dasatinib was not cost-effective if decision thresholds of £20,000 per QALY or £30,000 per QALY are used, compared with imatinib and nilotinib.

8






Nilotinib vs. dasatinib+2LNilo: $523,829/ LYICUR discounted:Imatinib+2LNilo vs. nilotinib: $282,515/ QALYImatinib+2LNilo vs. dasatinib+2LNilo: $662,144/ QALYNilotinib vs. dasatinib+2LNilo: $507,643/ QALYScenario 4Imatinib+2LNilo vs. nilotinib: $67,686/ QALYImatinib+2LNilo vs. dasatinib+2LNilo: $442,900/ QALYNilotinib vs. dasatinib+2LNilo: $183,929/ QALY

LYICUR discounted:Imatinib+2LNilo vs. nilotinib: £192,000/ QALYImatinib+2LNilo vs. dasatinib+2LNilo: £450,000/ QALYNilotinib vs. dasatinib+2LNilo: £345,000/ QALYScenario 4Imatinib+2LNilo vs. nilotinib: £46,000/ QALYImatinib+2LNilo vs. dasatinib+2LNilo: £301,000/ QALYNilotinib vs. dasatinib+2LNilo: £125,000/ QALY

BMT/ peripheral SCT

Breitscheidel 2008[19],Germany

CUA, €, 2005

Efficacy: RCT (IRIS), published studies QoL/Utilities: Patients (IRIS) EQ-5D (Imatinib); clinical panel: STG (rescaled, Lee et al. 1997[18], SCT)Costs: Red Book, DRG, EBM


ICUR discounted (undiscounted) Imatinib vs. MUD-SCT: $92,594/ QALY ($102,752/ QALY)

ICUR discounted (undiscounted) Imatinib vs. MUD-SCT: €69,764/ QALY (€77,410/ QALY)

“Imatinib is more costly but more effective (as measured in QALYs) over a 5-year time horizon. The resulting ICER of €77,410/ QALY is higher than commonly cited thresholds.”

Lee et al. 1997[18],IBMTR, NMDP U.S.

UA Efficacy: Published studies, clinician panelQoL/Utilities: 12 physicians: STGCosts: Not evaluated

Benefits: 3%, Costs: not evaluated

Unadjusted LE (in years)1)No BMT: 5.312)BMT within 1 year: 17.01 3)BMT at 1 to 2 years: 13.26 4)BMT at 2 to 3 years: 11.90 5)BMT at >3 years: 12.65 QALE discounted (in years)

“These results support the use of early unrelated donor bone marrow transplantation for most patients with CML.”

9






1)No BMT: 4.74 2)BMT within 1 year: 10.07 3)BMT at 1 to 2 years: 8.11 4)BMT at 2 to 3 years: 7.51 5)BMT at >3 years: 8.08

Lee et al. 1998[20],U. S.

CUA , US$, 1996

Efficacy: Meta-analysis of 7 RCTs, Lee et al. 1997[18]QoL/Utilities: Lee et al. 1997[18]Costs: Medical costs: accounting systems BWH, FHCRC; Medication costs: AWP, Red Book, pPatient records, published studies


ICUR discountedBMT vs. IFNα: $74,196/ QALY BMT vs. HU: $79,495/ QALY

ICUR discountedBMT vs. IFNα: $51,800/ QALY BMT vs. HU: $55,500/ QALY

“Unrelated donor transplantation for CML is expensive in absolute costs, but because it prolongs life substantially for some patients, the ratio of costs to effectiveness is in the range of other well-accepted medical interventions.”

Skrepnek and Ballard 2005[21],U.S.

CEA U.S.$, 2004

Efficacy: RCTs (incl. IRIS), published studiesQoL/Utilities: Not evaluatedCosts: Fee Reference, Physicians' Fee and Coding Guide, average wholesale prices; expert clinical opinion; published data

Benefits: n.r., Costs: 5%

ICER discounted Markov cohort analysis: Imatinib vs. BMT: -$90,167/ survival Monte Carlo microsimulation: Imatinib vs. BMT: -$5,948/ survival

ICER discounted Markov cohort analysis: Imatinib vs. BMT: -$75,789/ survival Monte Carlo microsimulation: Imatinib vs. BMT: -$5,000/ survival

"In most cases, imatinib was both less costly and more efficacious than BMT in the 2-year treatment of CML."

Legend:AWP = Average wholesale prices; BMT = Bone marrow transplantation; BNF = British National Formulary; CEA = Cost-effectiveness

analysis; CML = Chronic myeloid leukemia; CUA = Cost-utility analysis; Curtis = Unit Costs of Health and Social Care; DRG = Diagnosis

10

Related Groups; EBM = German Common Tariff Scale (Einheitlicher Bewertungsmaßstab der kassenärztlichen Bundesvereinigung); EQ-5D

= EuroQol 5D questionnaire; FASS = Pharmaceutical specialties in Sweden; FCMLG = French Chronic Myeloid Leukemia Study Group; HU

= Hydroxyurea; IBMTR = International Bone Marrow Transplant Registry; ICER = Incremental cost-effectiveness ratio; ICUR = Incremental

cost-utility ratio; IFNα = Interferon-alpha; IRIS = International Randomized Study of Interferon and STI571; LE = Life expectancy; LY =Life

year; LYS =Life years saved; LDAC = Low-dose cytarabine; MIMS = Monthly Index of Medical Specialties; MUD-SCT = Allogeneic stem cell

transplantation with a matched unrelated donor; NHS = National Health Service; NMDP = National Marrow Donor Program; NHS = National

Health Service; PFS = Progression-free survival; QALE = quality-adjusted life expectancy; QALY = Quality-adjusted life year; QoL = Quality

of Life; RCT = Randomized Controlled Trial; RMB = Renminbi (Chinese currency); SCT = Stem cell transplantation; SUHT = Southampton

University Hospitals NHS Trust; TTO = time-tradeoff; U.K. = United Kingdom; U.S. = United States of America; VAS = Visual analogue scale;

vs. = versus; WAC = Wholesale acquisition costs; € = Euro; $ = U.S. Dollar; £ = Pounds sterling; & = And; 2LNilo = Second-line nilotinib

Footnotes (corresponding to table II)

When the index year used for the economic evaluation was not stated, it was estimated to be the year prior to publication.

Economic results were transferred into 2011 US Dollar for comparability. This was done in two steps:

¥ Converting the currency into US Dollar of the same year using Purchasing power parity (PPP) rates

11

(http://stats.oecd.org/Index.aspx?datasetcode=SNA_TABLE4)[2], for example, € 2004 transformed to US$ 2004

† Converting US Dollar from step one into US Dollars 2011 (ftp://ftp.bls.gov/pub/special.requests/cpi/cpiai.txt)[1].

12

Reason study exclusion last step

Table III: Reason for study exclusion (1)

Reference Reason for Exclusion

1. Anonymous

2003[22]

Study Type

2. Anstrom 2004[23] Model*

3. Baccarani 1992[24] Publication Type

4. Bottemann

2010[25]

Publication Type

5. Garside 2002[26] Model

6. Goldman 2005[27] Publication Type

7. Gratwohl 2007[28] Publication Type

8. Hoyle 2011[29] Study Type

9. Kasteng 2007[30] Study Type

10. McGlave 1992[31] Study Type

11. Redaelli 2003[32] Study Type

12. Roeder 2008[33] Study Type

13. Roeder 2006[34] Study Type

14. Shen 2009[35] Publication Type

15. Simon 2006[36] Model

16. Stephens 2010[37] Intervention

17. Taylor 2009[38] Publication Type

*Used as Background in Reed 2004[8]and 2008[11]

13

Table IV: Definition: Reason for study exclusion (2)

Reason Explanation

Intervention Does not evaluate a treatment for CML

Model No Model or not sufficiently structured: based

on a decision-analytic model or any other type

of mathematical healthcare model evaluating

therapeutic interventions for

CML

Study Type Purely descriptive studies or studies using

models only as an illustration or in a tutorial

were excluded

Publication

Type

No full texts available (e.g., only abstract, or

comment or letter)

14

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