Insight

FDA's Accelerated Approval Program: Accelerated Pathway for Reform?

Reforms to FDA Accelerated Approval Program could include new confirmatory study requirements.

Larry Kocot

Larry Kocot

Principal, Advisory, KPMG LLP

+1 202-533-3674

Tracey McCutcheon

Tracey McCutcheon

Specialist Director, Advisory, KPMG US

+1 202-533-5380

Ross White

Ross White

Director, Advisory, KPMG US

+1 202-533-3691

The Accelerated Approval Program was established by the Food and Drug Administration (FDA) in 1992 to allow for earlier approval of drugs that treat serious conditions and that fill an unmet medical need based upon a surrogate endpoint that is likely to predict a clinical benefit. The pathway was codified in 2012 with the enactment of the Food and Drug Administration Safety and Innovation Act (FDASIA). Drugs approved through the Accelerated Approval Program must address a serious condition “associated with morbidity that has substantial impact on day-to-day functioning,” offer “meaningful advantage over available therapy,” and demonstrate an “effect on an endpoint that is reasonably likely to predict clinical benefit.”

Sponsors of drugs approved through the Accelerated Approval Program must produce data establishing that the surrogate endpoint or intermediate clinical endpoint is “reasonably likely” to predict a drug’s intended clinical benefit and agree to conduct post-marketing confirmatory stud(ies) to verify such benefit. Failure to demonstrate clinical benefit may result in withdrawal of approval or adjustment of the labeled indication. As of early 2021, roughly half of drugs receiving accelerated approvals have gone on to receive full approval as safe and effective by the FDA with a median time to approval of 3.2 years. An additional 6.3% of approvals have been withdrawn by the drug sponsor and 44.3% have been on the market a median of 1.9 years.

Growing concerns about drugs receiving accelerated approval without sufficient clinical benefit evidence or remaining on the market for extended periods of time without demonstrating benefit in post-approval studies, have led to increased calls for reforms to the Program. On March 7, 2022, House Energy and Commerce Committee Chair, Frank Pallone (R-NJ), introduced the Accelerated Approval Integrity Act of 2022 (H.R. 6963). The following day, the Committee’s Ranking Member, Congresswoman Cathy McMorris Rodgers (R-WA), introduced an alternative bill, Accelerating Access for Patients Act of 2022 (H.R. 6996).