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 post-approval 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 Some have argued that this “clinical-trial cliff” results from losing a substantial number of good drugs to outdated and impractical clinical-trial designs.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.