National Academy of Medicine

Innovation in Development, Regulatory Review, and Use of Clinical Advances: A Vital Direction for Health and Health Care

By Michael Rosenblatt, Christopher P. Austin, Marc Boutin, William W. Chin, Steven K. Galson, Amgen; Sachin H. Jain, Michelle McMurry-Heath, Samuel R. Nussbaum, John Orloff, Steven E. Weinberger, and Janet Woodcock
September 19, 2016 | Discussion Paper
About the Vital Directions for Health and Health Care Series

Vital DirectionsThis publication is part of the National Academy of Medicine’s Vital Directions for Health and Health Care Initiative, which called on more than 100 leading researchers, scientists, and policy makers from across the United States to provide expert guidance in 19 priority focus areas for U.S. health policy. The views presented in this publication and others in the series are those of the authors and do not represent formal consensus positions of the NAM, the National Academies of Sciences, Engineering, and Medicine, or the authors’ organizations.
Learn more: nam.edu/VitalDirections

This paper describes issues and challenges in inventing and regulating new medicines, vaccines, and devices and in integrating these advances into clinical practices as rapidly as appropriate and possible. It describes the landscape of discovery and invention, evaluation of efficacy and safety, determination of value, and postapproval surveillance and identifies windows of opportunity. It provides the rationale for markedly enhanced patient input throughout the process from target identification to decisions regarding insurance coverage. It describes the role of academe–industry collaboration in speeding the translation of research findings into health benefits and emphasizes the opportunity for medical education at multiple levels to realize the value of therapeutic innovations to society. Finally, it offers high-priority recommendations.

Context and Types of Opportunities

The pharmaceutical and biotechnology sectors experienced considerable challenges during the first decade of the 21st century. Stagnant research and development (R&D) productivity and the slow pace and high cost of drug development led many to argue for new approaches to discovery, manufacturing, development, and commercialization of new products to meet patients’ needs. Estimated costs for bringing a new drug to market through the research, development, and regulatory processes, may be as much as $2.6 billion, a substantial increase over the previous decade. The complexities of the analytics and cost attributions present challenges that are sources of active discussion, but there is no question that the costs are substantial. Furthermore, about 85% of therapies fail through early clinical development, and only half those surviving to Phase III will be approved. Some have argued that this “clinical-trial cliff” results from losing a substantial number of good drugs to outdated and impractical clinical-trial designs. Those challenges are forcing all sectors (industry, regulators, academe, government agencies, and patient advocacies) to evaluate opportunities to replace traditional drug-development paradigms with newer and more efficient models. Read more >>

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Note

Disclaimer: The views expressed in this paper are those of the authors and not necessarily of the authors’ organizations, the National Academy of Medicine (NAM), or the National Academies of Sciences, Engineering, and Medicine (the National Academies). The paper is intended to help inform and stimulate discussion. It is not a report of the NAM or the National Academies. Copyright by the National Academy of Sciences. All rights reserved.