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Executive Summary

NDA applicants will not be "forced" to use the accelerated approval mechanism, FDA said in the preamble to the final rule published in the Dec. 11 Federal Register. In response to comments from the Pharmaceutical Manufacturers Association expressing concern that applications could be "forced" to undergo accelerated approval, FDA said it "does not agree. ...A sponsor that wishes the application to be considered under the traditional approval process may request and receive such consideration." "It is true, however, that some proposed surrogate endpoints would not be considered acceptable bases for approval without assurance that the clinical studies to show clinical benefit will be conducted," FDA notes. PMA objected to the proposed rule, published April 15 ("The Pink Sheet" April 20, p. 8), in part because the association felt that the reg intrudes into postmarketing studies, an area traditionally subject to case-by- case negotiations between sponsors and FDA ("The Pink Sheet" July 27, p. 8). The final rule is essentially unchanged from the April 15 proposal, despite negative comments on the original proposal from industry. However, by emphasizing the voluntary nature of the rules, FDA is making an effort to defuse some of the sharpest criticism. For example, PMA argued in its July comments that FDA lacked the statutory authority to streamline the withdrawal process for drugs that fail to show efficacy in postmarketing trials. Maintaining that it has such authority, FDA noted that "applicants will have agreed that these withdrawal procedures apply to the drug for which they seek approval; applicants objecting to these procedures may forego approval under these regulations and seek approval under the traditional approval process." FDA also sought to damp criticism by emphasizing the limited scope of the reg. While comments focused on the fairly broad list of diseases eligible for accelerated approval, the final reg emphasizes the "limited group of circumstances" in which they will apply; that is, when the only available data involve an unvalidated surrogate marker or the drug has a side effect requiring limited distribution. Criticism of the accelerated approval proposal was not limited to industry. FDA received negative comments on aspects of the proposal from sources as diverse as the American Society of Clinical Oncology ("The Pink Sheet" July 6, T&G-9) and FDA Neuropharmacological Drug Products Director Paul Leber, MD ("The Pink Sheet" June 29, p. 12). FDA received 54 comments on the proposal, and "many comments posed specific questions and raised important concerns," the notice states. FDA's decision to publish the reg without substantially changing any of its provisions appears, in part, to be a reflection of the strength of several patient advocacy groups. The strongest support for the proposed reg has come from patient groups. The final reg does clarify the time frames for promotional preclearance requirements for accelerated approval drugs ("The Pink Sheet" April 20, p. 10). All materials planned for distribution within 120 days of approval need to be submitted to the agency for review during the preapproval period, the reg states. Postlaunch materials need to be submitted 30 days prior to distribution. FDA also has added a new section of the reg dealing with termination of the postmarketing requirements, although the agency did not adopt Merck's suggestion that it agree to act upon all postmarketing accelerated approval submissions within 180 days ("The Pink Sheet" Aug. 10, T&G-5). The agency, however, did say it "believes most reviews will be completed and action taken within 180 days." The accelerated approval initiative was part of Vice President Quayle's Council on Competitiveness FDA reform plan announced in November 1991 ("The Pink Sheet" Nov. 18, 1991, p. 5). The reg had been under development at FDA for a year prior to the Competitiveness Council announcement.

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