Dissent in the research community regarding this decision was muted but real. Concerns were raised that despite specific labeling that identified the drug as useful only in patients who had AIDS as evidenced by PCP or severe ARC, even asymptomatic patients might demand and physicians acquiesce to treatment with AZT, thereby risking major adverse effects in patients who might expect at least several healthy years if untreated. In contrast, others believed that all patients with AIDS, not only those who had PCP, were equally in danger of death and should receive the drug and that the labeling was thus too narrow. In an unprecedented program, Burroughs-Wellcome announced plans to collect postlicensing data on 20,000 patients receiving AZT to lessen the possible danger of widespread use of a drug on the basis of minimal data on toxicity and efficacy.

The Rise of Advocacy

As AZT quickly made its way from test tube to pharmacy in 1987, two other major developments occurred: AIDS activists, most notably represented by the AIDS Coalition to Unleash Power (ACT-UP), became increasingly visible, and the primary federal mechanism for the conduct of clinical trials, the AIDS Clinical Trials Group of the National Institute of Allergy and Infectious Diseases, expanded rapidly. Advocacy by grass-roots organizations in the gay community had been strong almost from the beginning of the epidemic. Initially focusing on issues of discrimination and access to health care, such groups became increasingly involved with issues of experimental treatments.

Advocacy groups made the FDA their prime focus, and from 1987 to 1989 they steadily and forcefully challenged the FDA to widen and speed the availability of drugs for AIDS. In addition, the Reagan administration, with its general philosophy of deregulation, became a sympathetic (if ironic) partner in the push toward greater consumer choice in AIDS drugs. Together, patient activism, a political climate favoring deregulation, and the exigency of an epidemic disease drove the most dramatic changes in drug regulation since the Kefauver-Harris legislation of 1962.

Initially, the changes were largely modifications of existing programs. As noted above, prior to 1987 physicians treating patients with life-threatening disease for which they had exhausted all licensed therapeutic options, could (on a case-by-case basis) obtain therapeutic agents that were still under investigation. Several variations on the basic process evolved, but terminology and procedures were ambiguous, and use of the various processes was sporadic and not well known in the medical community. Several attempts had been made to codify the ''compassionate use of drugs," but regulation by the FDA only occurred in 1987 under pressure from AIDS activists.



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