Accelerated Approval

Key facts

Created

1992, by FDA regulation

Current legal basis

Section 506(c) of the FD&C Act, codified by FDASIA in 2012

Regulations

21 CFR Part 314 Subpart H (drugs), 21 CFR Part 601 Subpart E (biologics)

Required after approval

Confirmatory trials to verify clinical benefit

Major reform

Food and Drug Omnibus Reform Act of 2022 (FDORA)

What is accelerated approval?

Traditional approval rests on evidence that a drug improves how patients feel, function or survive, or on an endpoint already validated to predict that. Accelerated approval lets the FDA act earlier, based on a surrogate endpoint reasonably likely to predict clinical benefit, such as tumor shrinkage, or on an intermediate clinical endpoint.

The pathway began in 1992, largely in response to the HIV/AIDS epidemic, and has been used most heavily in oncology. It is an approval, not a designation: products approved this way are fully marketed, but with a legal obligation to confirm their benefit.

How accelerated approval works

The pathway runs in four stages:

• Eligibility: the product treats a serious condition and offers a meaningful advantage over available therapy.
• Endpoint agreement: the sponsor and the FDA agree that the surrogate or intermediate endpoint is reasonably likely to predict clinical benefit.
• Approval with conditions: the FDA requires postmarketing confirmatory trials; since FDORA it can require those trials to be underway before approval and progress reports every 180 days.
• Confirmation or withdrawal: if trials fail to verify benefit, or are not conducted with due diligence, the FDA can withdraw approval through expedited procedures.

Why accelerated approval matters

Accelerated approval can bring revenue years earlier, but it adds a risk that traditional approvals do not carry. Analysts should track confirmatory trial status, because a negative result can narrow a label or remove a product from the market.

The pathway has drawn scrutiny. The 2021 accelerated approval of aducanumab (Aduhelm) for Alzheimer's disease was widely criticized, and the FDA withdrew approval of hydroxyprogesterone caproate (Makena) in 2023 after its confirmatory trial failed to show benefit.

Accelerated approval vs expedited designations

Fast track and breakthrough therapy are designations that change how the FDA works with a sponsor during development, and priority review shortens the review clock. Accelerated approval is the only one of the four that changes the evidence basis for approval, and a single product can use several of them.