© ISTOCK.COM/RHUI 1979; SYLVERARTSIt was October 1988, and AIDS had reached epidemic proportions in the U.S. A crowd gathered in front of the US Food and Drug Administration (FDA) in Rockville, Maryland. “Forty-two thousand dead from AIDS,” the protestors chanted. “Where was the FDA?” The Centers for Disease Control and Prevention later concluded that more than 62,000 people in the U.S. had died from AIDS by the end of 1988.
The protestors, organized by the advocacy group ACT-UP, demanded that the FDA abandon placebo-group requirements in clinical studies testing AIDS drugs and radically speed access to any therapies that showed promise.
A week after the protest, the FDA announced that it would begin to consider approving drugs for life-threatening illnesses based on Phase 2 trial results. Drug companies could still be obligated to carry out additional trials after approval. Four years later, the agency added to this framework the concept of surrogate endpoints—predictors of a drug’s efficacy such as change in tumor size. Such indicators can be assessed more quickly than traditional endpoints such as disease recovery or death. Under the resulting accelerated-approval program, officially launched in 1992, drugs that demonstrate these indirect signals of ...
