agency was able to use its bully pulpit to powerful effect in its interactions with sponsors (IOM Staff Notes, 2005–2006). However, consumer organizations, legislators, scientists, and others who have called for strengthening and clarifying FDA regulatory authority have provided numerous examples of cases where the agency was unable to effect desired changes. The committee asserts that the bully pulpit route leaves potentially critical regulatory action vulnerable to a subjective and highly variable process of exercising individual or agency influence, and to the vicissitudes of changing attitudes toward regulation. That is why FDA’s authorities must be clarified and strengthened to empower the agency to take rapid and decisive actions when necessary and appropriate.
Two Exceptions in FDA’s Regulatory Authority
Pediatric drugs and accelerated approval drugs provide two important incentive mechanisms with which to circumvent the imbalance in regulatory authority pre- and postapproval, and may be instructive as models for strengthening the statutory authorities available to FDA. The FDA Modernization Act of 1997 included patent exclusivity provisions as an incentive for sponsors who conducted studies of approved drugs in pediatric populations, and the 2002 Best Pharmaceuticals for Children Act renewed those incentives. That legislation exemplifies the “carrot” approach to motivating conduct and completion of studies: no study, no extended period of exclusivity. The “stick” approach to enforcing study commitments, which has not worked so well, is illustrated by accelerated approvals on the basis of surrogate endpoints (e.g., for cancer drugs) “which allows products to be used in nonresearch clinical care settings before they have been reliably established to have a favorable benefit-to-risk profile” (Fleming, 2005). Here again, however, FDA’s authority to enforce these commitments rests on withdrawing approval if the company does not complete the requisite studies and the high value of such therapeutic agents makes withdrawal undesirable. FDA’s authority to enforce should be made explicit, as it is for accelerated approvals, and the agency should also be given additional tools to enforce that authority. The power to withdraw is not a realistic tool as demonstrated by an FDA study of 8 drugs granted accelerated approvals. The average length of time for completion of required validation studies was 10 years, and it is unclear what FDA is able to do if studies are inconclusive (Fleming, 2005).