Accelerated approval lets the FDA approve a drug for a serious condition based on a surrogate endpoint (a lab or imaging marker reasonably likely to predict clinical benefit) rather than waiting for a hard outcome like survival. In exchange, the company must run confirmatory trials to verify the benefit.
Accelerated approval lets the FDA approve a drug based on a surrogate endpoint that is reasonably likely to predict clinical benefit, before that benefit is confirmed. Confirmatory trials are required after approval, and if they fail to verify benefit the FDA can withdraw the approval.
The trade-off
It gets important drugs to patients years earlier, but if the confirmatory trial fails, the FDA can withdraw the approval. That's a distinct, scheduled catalyst the market watches. Accelerated approval is one of several expedited pathways alongside priority review, Breakthrough Therapy, and Fast Track.
A lab value or imaging marker (e.g., tumor shrinkage) that is reasonably likely to predict real clinical benefit, used to support accelerated approval before long-term outcomes are known.
Can the FDA withdraw an accelerated approval?
Yes. If the required confirmatory trial fails to verify clinical benefit, the FDA can withdraw the approval.