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Remitting and Primary-Progressive Multiple Sclerosis: Effectiveness and Value

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Remitting and Primary-Progressive Multiple Sclerosis: Effectiveness and Value Disease-Modifying Therapies for Relapsing- Remitting and Primary-Progressive Multiple Sclerosis: Effectiveness and Value Draft Evidence Report November 22, 2016 Prepared for ©Institute for Clinical and Economic Review, 2016 ICER Staff and Consultants University of Washington School of Pharmacy Modeling Group Jeffrey A. Tice, MD Marita Zimmermann, MPH, PhD Associate Professor of Medicine Research Fellow University of California, San Francisco Pharmaceutical Outcomes Research and Policy Program, Department of Pharmacy Rick Chapman, PhD, MS University of Washington Director of Health Economics Institute for Clinical and Economic Review Elizabeth Brouwer, MPH Graduate Student Varun Kumar, MBBS, MPH, MSc Pharmaceutical Outcomes Research and Policy Health Economist Program, Department of Pharmacy Institute for Clinical and Economic Review University of Washington Anne M. Loos, MA Josh J. Carlson, PhD, MPH Senior Research Associate Associate Professor Institute for Clinical and Economic Review Pharmaceutical Outcomes Research and Policy Program, Department of Pharmacy Shanshan Liu, MS, MPH University of Washington Research Associate Institute for Clinical and Economic Review Matt Seidner, BS Program Manager The role of the University of Washington (UW) School of Institute for Clinical and Economic Review Pharmacy Modeling Group is limited to the development of the cost-effectiveness model, and the resulting ICER reports do not necessarily represent the views of the UW. Daniel A. Ollendorf, PhD Chief Scientific Officer Institute for Clinical and Economic Review David Rind, MD Chief Medical Officer Institute for Clinical and Economic Review Steven D. Pearson, MD, MSc President Institute for Clinical and Economic Review DATE OF PUBLICATION: November 22, 2016 We would also like to thank Margaret Webb for her contributions to this report. ©Institute for Clinical and Economic Review, 2016 Page i Draft Evidence Report – DMTs for RRMS and PPMS About ICER The Institute for Clinical and Economic Review (ICER) is an independent non-profit research organization that evaluates medical evidence and convenes public deliberative bodies to help stakeholders interpret and apply evidence to improve patient outcomes and control costs. ICER receives funding from government grants, non-profit foundations, health plans, provider groups, and health industry manufacturers. For a complete list of funders, visit http://www.icer- review.org/about/support/. Through all its work, ICER seeks to help create a future in which collaborative efforts to move evidence into action provide the foundation for a more effective, efficient, and just health care system. More information about ICER is available at http://www.icer- review.org About CTAF The California Technology Assessment Forum (CTAF) – a core program of ICER – provides a public venue in which the evidence on the effectiveness and value of health care services can be discussed with the input of all stakeholders. CTAF seeks to help patients, clinicians, insurers, and policymakers interpret and use evidence to improve the quality and value of health care. The CTAF Panel is an independent committee of medical evidence experts from across California, with a mix of practicing clinicians, methodologists, and leaders in patient engagement and advocacy. All Panel members meet strict conflict of interest guidelines and are convened to discuss the evidence summarized in ICER reports and vote on the comparative clinical effectiveness and value of medical interventions. More information about CTAF is available at https://icer- review.org/programs/ctaf/. ©Institute for Clinical and Economic Review, 2016 Page ii Draft Evidence Report – DMTs for RRMS and PPMS In the development if this report, ICER’s researchers consulted with several clinical experts, patients, manufacturers and other stakeholders. The following clinical experts provided input that helped guide the ICER team as we shaped our scope and report. None of these individuals is responsible for the final contents of this report or should be assumed to support any part of this report, which is solely the work of the ICER team and its affiliated researchers. For a complete list of stakeholders from whom we requested input, please visit: https://icer-review.org/material/ms-stakeholder-list/ Clinical Expert Reviewer David E. Jones, MD Assistant Professor of Neurology University of Virginia Health System Additional Clinical Expert Input Jeffrey Klingman, MD Chief of Neurology Kaiser Permanente Walnut Creek ©Institute for Clinical and Economic Review, 2016 Page iii Draft Evidence Report – DMTs for RRMS and PPMS Table of Contents Executive Summary ............................................................................................................................ ES1 1. Background ........................................................................................................................................ 1 1.1 Introduction ................................................................................................................................. 1 2. The Topic in Context .......................................................................................................................... 7 3. Summary of Coverage Policies and Clinical Guidelines ................................................................... 16 3.1 Coverage Policies ....................................................................................................................... 16 3.2 Clinical Guidelines ...................................................................................................................... 20 4. Comparative Clinical Effectiveness .................................................................................................. 22 4.1 Overview .................................................................................................................................... 22 4.2 Methods ..................................................................................................................................... 23 4.3 Results ........................................................................................................................................ 26 5. Other Benefits or Disadvantages ..................................................................................................... 57 6. Comparative Value ........................................................................................................................... 58 6.1 Overview .................................................................................................................................... 58 6.2 Cost-Effectiveness Model: Methods ......................................................................................... 58 6.3 Cost-Effectiveness Model: Results ............................................................................................. 69 6.4 Prior Published Evidence on Costs and Cost-Effectiveness of DMTs for MS ............................. 75 6.5 Summary and Comment ............................................................................................................ 80 References ........................................................................................................................................... 83 Appendix A. Search Strategies and Results .......................................................................................... 94 Appendix B. Ongoing Studies ............................................................................................................... 98 Appendix C. Comparative Clinical Effectiveness Supplemental Information .................................... 108 Appendix D. Network Meta-Analysis Methods and Results .............................................................. 132 Appendix E. Comparative Value Supplemental Information ............................................................. 150 Appendix F. Patient Survey Questions ............................................................................................... 185 ©Institute for Clinical and Economic Review, 2016 Page iv Draft Evidence Report – DMTs for RRMS and PPMS List of Acronyms Used in this Report AHRQ Agency for Healthcare Research and Quality BID Twice daily CDMS Clinically definite multiple sclerosis CI Confidence interval CIS Clinically isolated syndrome CNS Central nervous system CPI Consumer price index CrI Credible interval DMT Disease-modifying therapy DRG Diagnosis related group EDSS Expanded Disability Status Scale EQ-5D EuroQol five dimensions questionnaire FS Functional score HR Hazard ratio IM Intramuscular ITP Immune thrombocytopenic purpura IV Intravenous JC virus John Cunningham virus MS Multiple sclerosis MRI Magnetic resonance imaging MSFC Multiple Sclerosis Functional Composite MSIS-29 Multiple Sclerosis Impact Scale NNT Number needed to treat OR Odds ratio PML Progressive multifocal leukoencephalopathy PPMS Primary-progressive multiple sclerosis QALY Quality-adjusted life year QD Once daily QOD Once every other day QoL Quality of life REMS Risk evaluation and mitigation strategy RRMS Relapsing-remitting multiple sclerosis RR Rate ratio or risk ratio RRR Relative risk reduction SC Subcutaneous SF-12 12-item short form health survey SPMS Secondary-progressive multiple sclerosis TIW Three times a week USPSTF US Preventive Services Task Force ©Institute for Clinical and Economic Review, 2016 Page v Draft Evidence Report – DMTs for RRMS and PPMS Executive Summary An
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