Biologic Therapies for Treatment of Asthma Associated with Type 2 Inflammation: Effectiveness, Value, and Value-Based Price Benchmarks Evidence Report November 13, 2018 Prepared for ©Institute for Clinical and Economic Review, 2018 Authors: ICER Staff/Consultants* University of Colorado Skaggs School of Pharmacy Modeling Group† Jeffrey A. Tice, MD Jonathan D. Campbell, PhD Professor of Medicine Associate Professor University of California, San Francisco Department of Clinical Pharmacy Center for Pharmaceutical Outcomes Research Judith M. E. Walsh, MD, MPH Professor of Medicine Melanie D. Whittington, PhD University of California, San Francisco Research Instructor Department of Clinical Pharmacy Patricia Synnott, MALD, MS Senior Research Lead, Evidence Synthesis R. Brett McQueen, PhD Institute for Clinical and Economic Review Assistant Professor Department of Clinical Pharmacy Varun M. Kumar, MBBS, MPH, MSc Center for Pharmaceutical Outcomes Research Health Economist Institute for Clinical and Economic Review Samuel McGuffin, MPH Professional Research Assistant Ellie Adair, MPA Department of Clinical Pharmacy Program Manager Institute for Clinical and Economic Review Sean Sullivan, PhD Professor and Dean David Rind, MD, MSc University of Washington School of Pharmacy Chief Medical Officer Institute for Clinical and Economic Review Steven D. Pearson, MD, MSc President †The role of the University of Colorado Skaggs Institute for Clinical and Economic Review School of 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 UC. *None of the above authors have any conflicts None of the above modelers have any conflicts of interest to disclose of interest to disclose ©Institute for Clinical and Economic Review, 2018 Page i Evidence Report – Biologic Therapies for Treatment of Asthma DATE OF PUBLICATION: November 13, 2018 Jeff Tice served as the lead author for this report, with assistance from Judith Walsh. Jeff Tice, Judith Walsh, and Patricia Synnott led the systematic review and authorship of the comparative clinical effectiveness section. Varun Kumar was responsible for oversight of the cost-effectiveness analyses, and developing the budget impact model. The role of the University of Colorado 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 University of Colorado. Madeline O’Grady authored the section on coverage policies and clinical guidelines, with oversight from Ellie Adair. David Rind and Steven Pearson provided methodologic guidance on the clinical and economic evaluations. We would also like to thank Aqsa Mugal, Ariel Jurmain, Milon Waththuhewa, Laura Cianciolo, and Madeline O’Grady for their contributions to this report. ©Institute for Clinical and Economic Review, 2018 Page ii Evidence Report – Biologic Therapies for Treatment of Asthma 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. 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. The funding for this report comes from government grants and non-profit foundations, with the largest single funder being the Laura and John Arnold Foundation. No funding for this work comes from health insurers, pharmacy benefit managers, or life science companies. ICER receives approximately 20% of its overall revenue from these health industry organizations to run a separate Policy Summit program, with funding approximately equally split between insurers/PBMs and life science companies. Life science companies relevant to this review who participate in this program include: AstraZeneca, Genentech, GlaxoSmithKline, Regeneron, and Sanofi. For a complete list of funders and for more information on ICER's support, please visit http://www.icer- review.org/about/support/. About Midwest CEPAC The Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC) – 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. Midwest CEPAC seeks to help patients, clinicians, insurers, and policymakers interpret and use evidence to improve the quality and value of health care. The Midwest CEPAC is an independent committee of medical evidence experts from across the Midwest, with a mix of practicing clinicians, methodologists, and leaders in patient engagement and advocacy. All Council 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 Midwest CEPAC is available at https://icer-review.org/programs/midwest-cepac/. The findings contained within this report are current as of the date of publication. Readers should be aware that new evidence may emerge following the publication of this report that could potentially influence the results. ICER may revisit its analyses in a formal update to this report in the future. ©Institute for Clinical and Economic Review, 2018 Page iii Evidence Report – Biologic Therapies for Treatment of Asthma In the development of 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/asthma-stakeholder-list-2018/ Expert Reviewers Kenneth Mendez President and Chief Executive Officer The Asthma and Allergy Foundation of America Mr. Mendez personally owns stock in health care companies, but in an amount of less than $10,000. The AAFA receives greater than 25% of their funding to produce educational materials and for patient research through grants from pharmaceutical companies, including AstraZeneca, Genentech, GSK, Sanofi/Regeneron, and Teva. Matt Stevenson, PhD Professor of Health Technology Assessment University of Sheffield No relevant conflicts of interest to disclose, defined as more than $10,000 in health care company stock or more than $5,000 in honoraria or consultancies during the previous year from health care manufacturers or insurers. David Stukus, MD Associate Professor of Pediatrics Nationwide Children’s Hospital The Ohio State University College of Medicine No relevant conflicts of interest to disclose, defined as more than $10,000 in health care company stock or more than $5,000 in honoraria or consultancies during the previous year from health care manufacturers or insurers. ©Institute for Clinical and Economic Review, 2018 Page iv Evidence Report – Biologic Therapies for Treatment of Asthma Table of Contents Executive Summary ............................................................................................................................ ES1 1. Introduction ....................................................................................................................................... 1 1.1 Background .................................................................................................................................. 1 1.2 Scope of the Assessment ............................................................................................................. 4 1.3 Definitions .................................................................................................................................... 7 1.4 Insights Gained from Discussions with Patients and Patient Groups .......................................... 9 1.5 Potential Cost-Saving Measures in Asthma ................................................................................. 9 2. Summary of Coverage Policies and Clinical Guidelines ................................................................... 11 2.1 Coverage Policies ....................................................................................................................... 11 2.2 Clinical Guidelines ...................................................................................................................... 13 3. Comparative Clinical Effectiveness .................................................................................................. 18 3.1 Overview .................................................................................................................................... 18 3.2 Methods ..................................................................................................................................... 18 3.3 Results ........................................................................................................................................ 20 3.4 Summary and Comment ...........................................................................................................