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DIABETES, OTHER "SERIOUS" DISEASES ELIGIBLE FOR ACCELERATED APPROVAL UNDER PROPOSED RULE; QUAYLE, SULLIVAN, KESSLER ANNOUNCE UPCOMING REG PUBLICATION

Executive Summary

"Breakthrough" drugs for diabetes and other serious illnesses will be covered by FDA's proposed regulation for the accelerated approval of certain therapeutics, Commissioner Kessler told an April 9 press conference. Originally conceived as a vehicle for getting treatments for AIDS to the market more quickly, as currently drafted the proposed reg also will encompass treatments for serious but not necessarily life-threatening diseases, Kessler noted. The range is "broader than simply AIDS and cancer," Kessler said. In the proposed rule, "we talked about diseases that are not in the headlines," such as "cystic fibrosis, systemic lupus erythematosus...inflammatory bowel disease, [and] diabetes," the FDA commissioner said. Kessler's remarks came during a high-profile press conference on April 9. In addition to Kessler, Vice President Quayle, HHS Secretary Sullivan and HHS Assistant Secretary Mason commented on the progress of proposals put forth by Quayle's Competitiveness Council in November. The press conference focused on four topics: accelerated approval, the upcoming publication of the parallel track regulation, the contracting out of selected NDAs for external review and international harmonization of animal safety data requirements (see following stories). While accelerated approval was one of the suggestions put forth by the Council, an FDA-developed draft regulation already had been forwarded to HHS last fall. The proposal was an outgrowth of FDA's own exploration of how to speed up the review process, first via "conditional approval" and then by the agency's handling and approval of Bristol-Myers Squibb's Videx (didanosine, ddI) NDA as a second-line AIDS treatment ("The Pink Sheet" Nov. 18, 1991, p. 5). The accelerated approval reg will apply to "'breakthrough' drugs for patients with serious illnesses when a drug provides a meaningful therapeutic benefit over existing therapy," according to an HHS press release. The proposed regulation is expected to be published in the Federal Register April 15, FDA said. In addition to the diseases listed by Kessler, a fact sheet from the Vice President's office also lists Alzheimer's and depression as targets that would warrant accelerated approval. The spectrum of drugs eligible for accelerated review may become broader still. "We eventually want to open it up to the drugs that are going to impact all disease," Quayle said. "You have to start with the most important, the most life-threatening, the most serious," Kessler explained. "In the end we want to reduce the review time for all drugs," Kessler added. In a March 19 hearing before his Government Operations/Intergovernmental Relations subcommittee, Rep. Weiss (D-N.Y.) focused on the apparent give and take between FDA and the Vice President's Council on Competitiveness over the scope of the accelerated approval reg ("The Pink Sheet" March 23, p. 3). Weiss attempted to portray the development of the reg as a struggle between FDA's desire to focus exclusively on life- threatening conditions and the Administration's desire to include other chronic, serious diseases. Weiss suggested that the proposed reg was being held up by the Office of Management & Budget while the Administration tried to get FDA's support for expanding its scope. Weiss dismissed the April 9 press conference as "a politically timed restatement of what FDA already does under current law. This diversionary strategy seeks to mask the Administration's refusal to provide FDA with the resources it desperately needs to do its job." Quayle denied that the timing of the press conference was politically motivated: "We've been working on these proposals for quite some time....This is an announcement that had been planned for a long time." HHS' description of the accelerated approval proposal appears to have changed very little since Quayle and Kessler outlined it in November. The agency's October approval of ddl based on surrogate markers of AIDS progression remains "the prototype," Kessler said. Use of "surrogate endpoints" will be formalized, HHS said, so that "therapies would be approved at the earliest time in their development at which safety and effectiveness could be reasonably established." Sponsors will be required to commit to provide postmarketing clinical effectiveness data. The proposal will also include conditions for use of a "restricted distribution plan" and a "streamlined withdrawal process." Kessler stressed that accelerated approval would count as "full approval" and "will be reimbursed by the Health Care Financing Administration," thereby ensuring access to the medicines so approved. The FDA commissioner also used the forum to make comments on access to medicines that echoed remarks he made before the House Appropriations/Agriculture subcommittee in March ("The Pink Sheet" March 30, In Brief). Kessler is concerned that "we can speed up the process, we can get it out there, but unless people can afford those drugs, unless people have access to those drugs, what good is it?" Quayle repeated his November comments that accelerated approval would result in significant savings to the consumer. "I have talked to [drug companies] privately, I have talked to them at public gatherings, and I have said...that you had better pass on these savings to the consumer," the Vice President said. The companies, he warned, "better listen." Kessler was asked to name specific drugs that might be eligible for accelerated approval. He noted that FDA's Anti-Viral Drugs Advisory Committee will review clinical data for Videx on April 20 and then will review the safety and efficacy of Hoffmann- La Roche's Hivid (ddC, zalcitabine) on April 20-21, looking at data based on surrogate markers ("The Pink Sheet" March 16, In Brief). In the oncology area, Kessler said there are "a host of promising agents" including Bristol's taxol. "We have our researchers doing some of the important underlying analysis" of taxol, Kessler said. Clinical Pharmacology Division Director Jerry Collins, PhD, "is doing the pharmacokinetics on the drug at FDA," Kessler said. "We are very much a part of the drug development process."

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