The Future of Drug Safety:

Promoting and Protecting the Health of the Public

Institute of Medicine

Phone News Conference

September 22, 2006

Opening Statement

by

Sheila Burke

Deputy Secretary and Chief Operating Officer

Smithsonian Institution

and

Chair, Committee on the Assessment of the U.S. Drug Safety System

Good afternoon.  Thank you for joining us for the release of our report to the Food and Drug Administration on the assessment of the U.S. drug safety system.  This report was also supported by the Centers for Medicare and Medicaid Services, the Agency for Healthcare Research and Quality, the National Institutes of Health (in the Department of Health and Human Services), and the Department of Veterans Affairs.  My fellow committee members Professor Alta Charo and Dr. Bruce Psaty are joining me by phone today.

The focus of this report was solely on prescription drugs and on the drug safety system, in particular the functioning of FDA's Center for Drug Evaluation and Research, which is responsible for drug review, approval, and regulation.

The report recognized at the outset that it is impossible to think about safety independent of efficacy, and that the two must be considered together throughout the life cycle of a drug -- from drug discovery until the end of useful product life.  Drugs are approved on the basis of risk-benefit determinations made by FDA, but those determinations are based on clinical trials with carefully selected participants and under controlled conditions.  The real-life use of drugs is often quite different -- a drug tested in a few hundred or thousand people is prescribed and used by millions often for longer periods and in conjunction with other drugs or supplements.  That is why approval does not signify the end of uncertainty about a drug, and continued monitoring is necessary after approval.  Most stakeholders in the drug safety system are aware of the need for continued attention to a drug's risk-benefit profile.  However, the committee found an imbalance in the regulatory attention and resources available before and after a drug's approval.  Staff and resources devoted to pre-approval are substantially greater than those available for post-approval functions.  Many of the committee's recommendations are intended to bring the strengths of the pre-approval process to the post-approval process, to ensure ongoing attention to a drug's performance.

Anyone who has followed the drug safety saga of the last several years has surely noticed that information has emerged -- both in the media and in government report -- about poorly handled scientific disagreement, a lack of collaboration among divisions, an appearance of interdisciplinary tension, a perception of inappropriate management expectations, and so on.  The committee also took notice of that. 

FDA's regulatory authorities are derived from a statute that has been amended numerous times, yet requires strengthening and clarification to allow the agency the flexibility to regulate increasingly complex drugs.  In its discussion of FDA's ability to regulate, the committee was cognizant of the fact that the outcomes of regulation are not paper documents but the health of living, breathing patients.  Delaying approval until complete certainty is reached, or withdrawing a drug once safety problems arise are often not realistic options, yet they reflect the largely all-or-nothing nature of FDA regulatory authorities.  This committee recommended that FDA be given a tool kit of regulatory options it can apply as appropriate and necessary at any time in the life cycle of a drug, and clarified authority to enforce sponsor compliance with restrictions or limitations on marketing imposed at or after the time of approval. 

The commitment of public servants, the concern of Congress, the advocacy of consumer organizations, the interest of industry, among others, are not enough to transform the drug safety system in the ways outlined by the committee's suite of recommendations.  We trust the FDA to protect the public's health and that is why we have recommended substantial added authority, responsibilities, and financial investment for FDA.  

The committee is grateful to have had the opportunity to be of assistance to FDA, and hopes that the agency finds the report useful.  This concludes my opening remarks.  My colleagues and I will be happy to take your questions now.