Accelerated Approval
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Accelerated Approval lets FDA approve a drug for a serious condition based on an effect on a surrogate or intermediate clinical endpoint reasonably likely to predict clinical benefit, rather than waiting for confirmed clinical benefit itself, when the drug fills an unmet need. The applicant must then complete postmarketing confirmatory trials verifying the anticipated clinical benefit.
The tradeoff is speed for uncertainty: approval comes sooner because it rests on a marker believed to predict benefit rather than a demonstrated clinical outcome, so accelerated approval carries an explicit obligation — reinforced by 2023 statutory changes strengthening FDA’s enforcement tools — to conduct confirmatory trials with due diligence.
If confirmatory trials fail to verify clinical benefit, or the applicant fails to conduct them with due diligence, FDA can withdraw the approval through an expedited procedure — a mechanism FDA has used more actively in recent years after criticism that some accelerated approvals lingered on the market for years without confirmatory data.
- —Approval based on a surrogate/intermediate endpoint reasonably likely to predict clinical benefit
- —Requires postmarketing confirmatory trials to verify the anticipated benefit
- —FDA can withdraw approval through an expedited procedure if confirmation fails
- —Distinct from Priority Review, which shortens the review clock rather than the evidence bar
21 CFR Part 314 Subpart H (drugs); 21 CFR Part 601 Subpart E (biologics); FD&C Act §506(c)
Frequently asked questions
What is Accelerated Approval?
Accelerated Approval lets FDA approve a drug for a serious condition based on an effect on a surrogate or intermediate clinical endpoint reasonably likely to predict clinical benefit, rather than waiting for confirmed clinical benefit itself, when the drug fills an unmet need. The applicant must then complete postmarketing confirmatory trials verifying the anticipated clinical benefit.
Which regulations cover Accelerated Approval?
21 CFR Part 314 Subpart H (drugs); 21 CFR Part 601 Subpart E (biologics); FD&C Act §506(c)