1
Introduction1

The U.S. Food and Drug Administration (FDA) today has a broad range of responsibilities, regulating fully 25 percent of the American economy, including 80 percent of the nation’s food supply and all drugs, devices, dietary supplements, animal drugs, cosmetics, biologics, and tobacco products. These responsibilities go far beyond those mandated in the agency’s originating legislation—the 1906 Pure Food and Drugs Act2 and the 1938 Federal Food, Drug, and Cosmetics Act.3 The agency was initially established to prevent the entry of adulterated products into the public market; it was from its inception grounded in principles of scientific study in support of its core mission of regulating consumable goods. That mission remains today; however, the agency has assumed a far more scientifically complex and international reach, with centers located around the world. In the face of rapid advances in medicine and biomedical science, the FDA faces pressure to keep pace with new technologies and develop the expertise necessary to regulate those technologies as they emerge. At the same time, however, stagnant funding levels and

1

While the Drug Forum conceived the idea for this workshop, this summary was prepared by the rapporteurs as a factual summary of the presentations and discussions that took place at the workshop. Statements, recommendations, and opinions expressed are those of individual presenters and participants, and are not necessarily endorsed or verified by the Drug Forum or the National Academies, and should not be construed as reflecting any group consensus.

2

Pure Food and Drug Act of 1906 (34 U.S. Stat. 768).

3

Federal Food, Drug, and Cosmetic Act (FDCA) (52 U.S. Stat. 1040).



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1 Introduction1 The U.S. Food and Drug Administration (FDA) today has a broad range of responsibilities, regulating fully 25 percent of the American economy, including 80 percent of the nation’s food supply and all drugs, devices, dietary supplements, animal drugs, cosmetics, biologics, and tobacco products. These responsibilities go far beyond those mandated in the agency’s originating legislation—the 1906 Pure Food and Drugs Act2 and the 1938 Federal Food, Drug, and Cosmetics Act.3 The agency was initially established to prevent the entry of adulterated products into the public market; it was from its inception grounded in principles of scien- tific study in support of its core mission of regulating consumable goods. That mission remains today; however, the agency has assumed a far more scientifically complex and international reach, with centers located around the world. In the face of rapid advances in medicine and biomedi- cal science, the FDA faces pressure to keep pace with new technologies and develop the expertise necessary to regulate those technologies as they emerge. At the same time, however, stagnant funding levels and 1 While the Drug Forum conceived the idea for this workshop, this summary was prepared by the rapporteurs as a factual summary of the presentations and discussions that took place at the workshop. Statements, recommendations, and opinions expressed are those of individual presenters and participants, and are not necessarily endorsed or verified by the Drug Forum or the National Academies, and should not be construed as reflecting any group consensus. 2 Pure Food and Drug Act of 1906 (34 U.S. Stat. 768). 3 Federal Food, Drug, and Cosmetic Act (FDCA) (52 U.S. Stat. 1040). 1

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2 ESTABLISHMENT OF REGULATORY SCIENCE FOR DRUG DEVELOPMENT staff turnover have increasingly hampered the agency’s ability to fulfill its regulatory mission. As discussed in this introduction, there has been much discussion regarding the urgency of evolving FDA to a robust and autonomous agency that has capacity to bridge gaps in knowledge and resources so that safe, effective drugs can be delivered to patients. Only recently, how - ever, have funds been requested specifically for the task of fortifying the science base behind FDA’s regulatory actions (FDA, 2010a). WORKSHOP PuRPOSE AND OBJECTIvES In its 2007 report The Future of Drug Safety: Promoting and Protecting the Health of the Public (IOM, 2007), the Institute of Medicine (IOM) iden- tified the need for an improved science base for drug evaluation within FDA, including both internal resources and extramural funding for col - laboration with academia. In that same year, the FDA Science Board,4 at the request of Congress, reported on the agency’s need for an enhanced science base, including infrastructure development, multisector collabo - ration, and an expanded workforce capable of addressing the rapidly evolving science of drug discovery and development. In 2008, the IOM Forum on Drug Discovery, Development, and Translation held a pub- lic workshop to explore the science of drug regulation, focusing on the gap between leading-edge technologies of drug development and FDA’s capacity to adapt its process of regulatory evaluation to these technologies (IOM, 2008). Together, the results of these efforts suggest a widening gap between scientific developments in areas relevant to FDA’s mission and its ability to address these innovations, as well as a lack of understanding among the public, policy makers, and the agency of what is required to fill this gap. To address these concerns, the IOM Drug Forum convened a public workshop on February 26, 2010, to examine the state of the science of drug regulation and consider approaches for enhancing the scientific basis of regulatory decision making. The workshop provided an oppor- tunity to explore the concept of regulatory science, examine how it can be used to improve regulatory decision making, and consider alterna- 4 The FDA Science Board is an advisory committee with the following mission: “The Board shall provide advice primarily to the Commissioner and other appropriate officials on specific complex and technical issues as well as, emerging issues within the scientific com - munity, in industry and academia. Additionally, the Board will provide advice to the Agency on keeping pace with technical and scientific evolutions in the fields of regulatory science, or formulating an appropriate research agendas; upgrading its scientific and research facilities to keep pace with these changes. It will also provide the means for critical review of Agency sponsored intramural and extramural scientific research programs” (FDA, 2010b).

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3 INTRODUCTION tive mechanisms and institutional frameworks for its development and application. Among the participants were experts in the science of drug regulation, as well as stakeholders in drug development and regulatory processes, including representatives of patient groups, academia, government, and industry. According to Gail H. Cassell, Vice President of Scientific Affairs and Distinguished Lilly Research Scholar for Infectious Diseases, Eli Lilly, and Drug Forum Co-Chair, the workshop had the following objectives: • stablish a clear definition of regulatory science, and engage the E public and the policy community in a discussion of its challenges and opportunities. • ncrease awareness of inadequate funding for regulatory science I and the impact on the development of new therapies on patients. • rticulate priorities and strategies for building or rebuilding the A infrastructure for regulatory science. A range of approaches and innovative mechanisms were considered, including fostering the scientific discipline of regulatory science, pro- moting closer collaboration between regulatory and academic research - ers, and developing a solid infrastructure to support regulatory science. Among the specific models discussed were regulatory science centers of excellence, innovative federal grant-making mechanisms, and medical education and professional development programs. ORgANIZATION OF THIS REPORT This report summarizes the presentations and discussions at the workshop, highlighting key themes and concepts for enhancing regula- tory science: • hapter 2 defines the concept of regulatory science in the context C of FDA. • hapter 3 explicates the need for regulatory science in today’s drug C development environment, including areas in which regulatory science could track emerging technologies. It also examines poten - tial negative consequences of deficiencies in regulatory science. • hapter 4 describes barriers to establishing a regulatory science C infrastructure. • hapter 5 explores a collaborative model for promoting regulatory C science and considers examples of past successes in similar fields. • hapter 6 examines ways to energize the public policy community C to support the development of regulatory science.

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 ESTABLISHMENT OF REGULATORY SCIENCE FOR DRUG DEVELOPMENT • hapter 7 elaborates on the desired outcomes of successful imple - C mentation of regulatory science. • hapter 8 summarizes next steps for enhancing regulatory science C offered during the workshop.