Food and Drug Administration

Azidothymidine (AZT) is a celebrated example of speedy FDA approval of a new drug. The unusually swift approval of AZT during the early years of the AIDS (ACQUIRED IMMUNE DEFICIENCY SYNDROME) epidemic led to the creation of a new FDA category, treatment investigational new drug (treatment IND), that established new procedures for more rapid and flexible drug approval.

The pharmaceutical company Burroughs Wellcome first presented AZT as a new drug to the FDA in June 1985. Public fear of AIDS had increased dramatically during the previous few years, as had protests by AIDS activists who complained of slow FDA movement with regard to promising new treatments. Keenly aware of the need for swift decision making in the face of the deadly AIDS disease, the FDA approved phase 1 clinical trials of the drug within one week of the initial application.

Phase 1 testing of AZT, between July and December 1985, was promising, and phase 2, involving 300 patients in placebo-controlled trials, began in February 1986. After six months, 19 of the 137 patients in the group taking the placebo had died, whereas only 1 of 145 in the group taking AZT had died. The results were encouraging enough for the FDA to forgo further testing. The group taking the placebo was switched to AZT, and phase 3 testing, traditionally the most important step in clinical trials, was deemed unnecessary.

In September 1986, the FDA authorized the treatment of patients who wanted access to AZT, even before it had given approval to the drug. By the time approval for general public use came in March 1987, some 4,000 patients had already been treated with AZT, and thus the drug was already potentially extending the lives of hundreds of people.

Taking an example from its handling of AZT, the FDA in May 1987 created the treatment IND classification to facilitate faster approval and wider distribution of promising new therapies for life-threatening diseases. The rapid approval of AZT also proved greatly encouraging to the pharmaceutical industry, which could now hope to bring AIDS drugs to market much more rapidly and at lower cost.