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Executive Summary

An FDA draft proposal for expediting the approval of drugs to treat life-threatening diseases is currently undergoing scrutiny at the Office of Management & Budget (OMB). The plan should get an expedited review at OMB, where the leadership includes Administration officials who directed FDA to develop the proposal. The executive director of Vice President Bush's Task Force on Regulatory Reform is Jay Plager, who also heads OMB's Office of Information and Regulatory Affairs. In an Aug. 24 memorandum explaining the proposal to HHS, FDA Commissioner Young said the plan "can be implemented effectively through a Federal Register notice that could be announced by the Vice President." Young said the "notice could take the form of either a statement of policy, a notice of proposed rulemaking, or an interim final rule" and "could be published by the end of September, if not sooner." Key features of the proposal include FDA-sponsored conferences before animal testing and at the end of Phase I; a "proactive" agency involvement in the process, prodding sponsors when necessary; FDA-conducted research; and greater reliance on postmarketing studies. "This approach embodies the genuine need many of us recognize to identify promising new drugs as early as possible and to facilitate their progress toward marketing approval to the fullest extent possible," the agency memo states. "This approach was used with great success during the development and review of AST," FDA observed. "We believe that formalizing and expanding upon it would implement the Vice President's direction to accelerate the drug approval process for new therapies to treat life-threatening illnesses." The proposal is expected to have the support of the Pharmaceutical Manufacturers Association. At a Sept. 8 meeting, PMA's Executive Committee recommended that the board endorse the plan. PMA is said to be particularly pleased that FDA determined the proposal does not require legislative authority. The agency memo states that FDA "can implement this new approach without legislation." Commissioner Young testified at a Senate hearing on AIDS drug development and review that statutory authority to require postmarketing studies might be useful to FDA ("The Pink Sheet" July 18, p. 9). However, the memo asserts, legislation is not necessary even for Phase IV authority "because, as with AST, sponsors are generally willing to conduct appropriate Phase IV studies voluntarily." By conducting end-of-Phase I study conferences to design more thorough Phase II studies, the agency plans to obviate the need for Phase III studies, the proposal states. "FDA's goal is to be able to reach a scientifically defensible decision based on the results of well-designed Phase II controlled clinical trials," according to the draft. "If a therapy is found to effectively treat a life-threatening disease for which no other therapy exists, it would not be appropriate to continue research into Phase III, once Phase II testing is found to be definitive." On the other hand, "poorly designed Phase II studies serve to retard the drug development process, as even equivocal results from such studies make it difficult to carry out the additional research needed to fully evaluate the drug," the proposal asserts. "In order to increase the likelihood that Phase II testing can provide definitive results, the sponsor would plan Phase II studies that are somewhat larger and more extensive than is currently the norm, including a mode for replication of key findings." For example, "to avoid missing an effect by using too little drug or to avoid studying a dose that proves toxic, it may be necessary to study several doses in the first formal trials, an approach that may require a larger study but can plainly save time," the draft explains. "However, it should be appreciated that if a drug has only minor or inconsistent effect, these limited beneficial results may be missed in this stage of clinical testing, even if the drug ultimately proves to be beneficial following more extensive Phase III trials." Young told HHS that enhanced FDA involvement "would include expediting the process by contacting the sponsor directly when clinical trials are not proceeding on schedule, and convening special meetings of FDA's advisory committees as necessary, rather than waiting for the next scheduled periodic meeting." The agency noted that Vice President Bush asked that the proposal include FDA authority to a factor risk-benefit analyses into its approval decisionmaking. "Clearly, for a life-threatening illness, a relatively high level of known risk and some uncertainty about potential risk from the drug can be acceptable in exchange for the improved survival provided by effective drug treatment for a condition that, left untreated, would result in death," the memo maintains.

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