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

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, however, have funds been requested specifically for the task of fortifying the science base behind FDA’s regulatory actions (FDA, 2010a).

In its 2007 report The Future of Drug Safety: Promoting and Protecting the Health of the Public (IOM, 2007), the Institute of Medicine (IOM) identified the need for an improved science base for drug evaluation within FDA, including both internal resources and extramural funding for collaboration 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 collaboration, 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 public 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 opportunity to explore the concept of regulatory science, examine how it can be used to improve regulatory decision making, and consider alterna-

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:

• Establish a clear definition of regulatory science, and engage the public and the policy community in a discussion of its challenges and opportunities.

• Increase awareness of inadequate funding for regulatory science and the impact on the development of new therapies on patients.

• Articulate priorities and strategies for building or rebuilding the infrastructure for regulatory science.

A range of approaches and innovative mechanisms were considered, including fostering the scientific discipline of regulatory science, promoting closer collaboration between regulatory and academic researchers, 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.

This report summarizes the presentations and discussions at the workshop, highlighting key themes and concepts for enhancing regulatory science:

• Chapter 2 defines the concept of regulatory science in the context of FDA.

• Chapter 3 explicates the need for regulatory science in today’s drug development environment, including areas in which regulatory science could track emerging technologies. It also examines potential negative consequences of deficiencies in regulatory science.

• Chapter 4 describes barriers to establishing a regulatory science infrastructure.

• Chapter 5 explores a collaborative model for promoting regulatory science and considers examples of past successes in similar fields.

• Chapter 6 examines ways to energize the public policy community to support the development of regulatory science.

• Chapter 7 elaborates on the desired outcomes of successful implementation of regulatory science.

• Chapter 8 summarizes next steps for enhancing regulatory science offered during the workshop.