FDA Guidance Advises RCTs for Oncology Drugs Seeking Accelerated Approval
Sponsors of new oncology drugs and biologics that aim to apply for Accelerated Approval (AA) should use a randomized controlled trial (RCT) design rather than a single-arm trial in most cases, the FDA advises in new draft guidance issued last week.
RCTs can be preferable to single-arm trials because they can offer the opportunity for longer-term follow-up that can meet the postmarket requirements of AA, the guidance says.
The 12-page guidance recommends sponsors conduct one RCT to support an AA application or two separate RCTs, one to support the application and another confirmatory trial to verify clinical benefit, the FDA says.
Additionally, the FDA recommends sponsors select an endpoint that can be evaluated earlier in the disease and trial and consider the investigational drug’s mechanism of action, the ability to reliably characterize the measurable disease and other context-reliant factors when choosing an endpoint.
The agency acknowledges there can be “significant concerns about the feasibility of a randomized controlled trial” in some cases, making a single-arm trial more appropriate. “Careful consideration should be taken in determining whether a single-arm trial is appropriate in a particular clinical and regulatory context,” according to the guidance, which advises sponsor to consult with the agency before making that decision.
The guidance also offers the FDA’s perspective on confirmatory trials, a key component of AA. It reminds sponsors to carry out these postmarket trials “with due diligence” and in line with the conditions set by the agency, including enrollment targets, the trial protocol and milestones, and the intended date of trial completion.
Comments on the draft guidance are due by May 26.
Read the draft guidance here: https://bit.ly/42BDQIY.
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