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‘Dangling’ Accelerated Approvals: US FDA Flags Concerns About ‘Marginal’ Response Rates

 
• By Sue Sutter

Failed confirmatory trials for PD-1/L-1 inhibitor indications raise questions about marginal response rates in single-arm trials used to support accelerated approval, Oncology Center of Excellence leaders say in NEJM article that explains why the agency is taking six of these indications to its Oncologic Drugs Advisory Committee.

Dangling mugs
US FDA's Oncology Center of Excellence has coined the term "dangling accelerated approvals" when confirmatory trials fail to verify clinical benefit but indications remain on the label. • Source: Shutterstock

Checkpoint inhibitor indications approved on the basis of marginal response rates in single-arm trials are a key focus of the US Food and Drug Administration’s class-wide evaluation of whether accelerated approvals should remain intact despite the failure of confirmatory trials.

In a New England Journal of Medicine article published online 21 April, Oncology Center of Excellence chief of medical oncology Julia Beaver and director Richard Pazdur write about the 10 “dangling” accelerated approval indications

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