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AIDS Treatment News
FDA Workshop on Clinical Trial Design, September 6-7 --
John S. James
August 4, 1995
AIDS Treatment News Issue #228, August 4, 1995

The FDA will hold a public workshop on the design of HIV clinical trials, September 6-7, 8:30 a.m. to 5:00 p.m., in Bethesda, Maryland. Following the workshop there will be a joint meeting of subcommittees of the Antiviral Drugs Advisory Committee and the National Task Force on AIDS Drug Development, on September 8. Both meetings will be held in the William H. Natcher Conference Center, at the National Institutes of Health, in Bethesda, Maryland.

Purpose -- and Background This workshop and the associated meetings will focus on dealing with the difficulties of designing "confirmatory" trials to use after accelerated approval of a drug. Under the accelerated approval system -- which was developed and implemented by FDA commissioner David Kessler -- new drugs can be approved based on "surrogate marker" indications of efficacy -- usually blood tests such as viral load or CD4 (T- helper) count. These treatments are then fully approved and can be marketed like any approved drug, and are reimbursed by insurance; the only difference is that, in return for approval based on blood tests, the accelerated approval regulations require that the company agree to perform confirmatory studies, after approval, to prove that the drug has real clinical benefit to patients. In practice, this has been taken to mean proving efficacy by "clinical endpoints" -- deaths or AIDS-defining opportunistic infections.

For various reasons, these confirmatory trials are turning out to be unusually difficult to do. For example, how do you get people to volunteer for a body-count study, to prove -- through their deaths or serious illnesses -- a drug which has already been generally accepted in the practice of HIV medicine? The goal of this workshop, according to its official announcement, "is to discuss the critical issues in the design and conduct of clinical confirmatory trials in HIV and to propose strategies to overcome identified obstacles so that new drugs can be made available more quickly and that information on how to best use them also be obtained without unnecessary delay." Deadlines Registration for the workshop is required by August 18, by fax to 301/443-9216 (with your name, organization if any, address, and phone number) or by Internet. Persons wishing to speak at the Advisory Committee meeting should telephone by August 25. After the meetings, persons may submit comments on the workshop until October 31, by mail or Internet. Because of the messiness of the additional details, we decided not to publish them here, but to refer interested persons to Heidi Marchand or Kimberly Miles, Office of AIDS and Special Health Issues, 301/443-0104.