Fully Enrolled Confirmatory Studies Can De-Risk Novel Accelerated Approval Endpoints – FDA Official

A novel endpoint for a cancer drug’s accelerated approval is more likely to get the go ahead from the US Food and Drug Administration if the sponsor has a fully accrued post-marketing study underway at the time of application, Oncology Center of Excellence Deputy Director Paul Kluetz said.

Kluetz noted this is one way in which an Oncology Center of Excellence project can help “de-risk” aspects of a drug’s development. In this instance he was discussing Project Frontrunner, which is pushing sponsors to study cancer treatments in earlier lines of care first, rather than starting with the most refractory setting, and pushing for more randomized trials for cancer accelerated approvals. In some cases, the agency envisions the same study being used for accelerated approval and post-market confirmation. (Also see "Cancer And Accelerated Approval: FDA To Crack Down On Single-Arm Trials, Refractory Disease Focus" - Pink Sheet, 10 June, 2022