By Healthcare Brew Staff
less than 3 min read
Definition:
Accelerated approval is a fast-tracked approval process offered by the FDA that pushes drugs for extremely serious or life-threatening conditions with unmet medical need to be approved through with less evidence than is normally required.
The history
The accelerated approval program was created in 1992 in response to the AIDs epidemic.
Traditional approvals require drugmakers to demonstrate their drug has clinical benefit. Accelerated approvals can be based on a surrogate endpoint—a measurement that’s likely, but not guaranteed, to predict clinical benefit.
Drugmakers must complete a confirmatory trial after the fast-tracked drug goes to market. If the trial shows clinical benefit, the FDA grants traditional approval.
Potential controversy
Some critics say this process bypasses necessary safeguards.
A January 2025 report from the Department of Health and Human Service’s Office of the Inspector General (OIG) found that for three of 24 sampled drugs, the FDA gave accelerated approval even though FDA reviewers or advisory committee members flagged the cases for not following protocol.
One controversial example, not included in that review, is Sarepta Therapeutics’s Elevidys gene therapy for Duchenne muscular dystrophy.
Two patients died of acute liver failure—a known potential side effect—after taking the drug, one announced in March 2025 and one in June 2025.
The deaths came after Peter Marks, the FDA’s former director of the Center for Biologics Evaluation and Research, overruled his staff to give the drug accelerated approval in June 2023, then full approval in June 2024.
Many Duchenne advocates and doctors defended accelerated approvals despite this tragedy. The FDA required Sarepta to significantly update the drug’s safety label.
