Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders Workshop Summary (2015) / Chapter Skim
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1 Introduction and Overview
1 Introduction and Overview
Pages 1-12
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These conditions generate great human suffering and impose a tremendous economic load. According to 2014 estimates from the Society for Neuroscience, nearly 100 million Americans suffer from nervous system disorders, and associated annual expenses exceed $760 billion (Choi et al., 2014)
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Representatives from academia, government agencies, patient groups, and industry gathered to share information and viewpoints, and to brainstorm about budget-neutral policy changes that could help widen the pipeline toward drugs that address unmet needs for CNS disorders. Pull Incentives: Improving Market Protections and Regulatory Processes This workshop concentrated on "pull" incentives that might maintain and strengthen private-sector investment in CNS R&D innovation by increasing market returns.
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The incentives discussed at the workshop do not require significant public funding. For example, additional market protection for breakthrough drugs that address unmet medical needs, adaptive trial design in which companies have the ability to modify ongoing studies, and conditional regulatory approval of drugs that demonstrate substantial improvements early during clinical development.
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. To increase efficiency in clinical trials, movements are afoot to create standing clinical trial platforms that are global in nature and that characterize patients the same way, so drugs can be run through the platforms more quickly in Phase II and Phase III trials, said George Vradenburg, chairman and founding board member of USAgainstAlzheimer's, and Janet Woodcock, director of the Center for Drug Evaluation and Research, Food and Drug Administration (FDA)
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Chapter 2 reviews current market protections and offers possible ways to extend existing legislation. Chapter 3 provides an overview of current regulatory pathways, and the challenges and opportunities in this sector.
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According to the Milliman and American Psychiatric Association 2014 report, "patients with behavioral health conditions cost an estimated $525 billion in health care expenditures annually" (Melek et al., 2014, p.
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. The number of publicly visible clinical CNS programs in 11 large pharmaceutical companies dropped by 50 percent between 2009 and 2011 (Choi et al., 2014)
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. Historically, large companies have played an especially important role in optimizing lead molecules and thus turning them into drugs; these outfits have also led in funding the large Phase III clinical trials that establish drug safety and efficacy, an essential step in bringing drugs to market.
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Current animal models and laboratory assays do not always predict therapeutic efficacy; the human brain is inaccessible to direct study; and robust biomarkers are scarce (IOM, 2013b)
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The objective of this workshop was for participants to discuss approaches for incentivizing R&D that will produce therapies that target unmet medical needs and significantly improve lives in the area of CNS diseases by strengthening market protections and regulatory processes. The intent was not to encourage development of drugs whose structures closely resemble existing agents and that act by the same mechanism of action ("me too" drugs)
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To address this issue, a few workshop participants emphasized that patients have a critical role in conversations about how to balance uncertainty and potential benefits when considering how and when to use these options. Patient Benefit and Advocacy • Several participants noted the importance of involving patients and caregivers early on in discussions about strategies that might increase incentives for CNS drug development.
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