The US Food and Drug Administration (FDA) aimed to address some of the criticisms of its accelerated approval program with new draft guidance that expresses a preference for using randomized controlled trials (RCTs) to support the approval of oncology drugs in the program. But in public comments, stakeholders asked for more details on when it is appropriate to pursue a single-arm design.
Commenters also called on the agency to provide more specifics on surrogate endpoints and the use of real-world data and evidence.
The draft guidance, issued in March 2023, outlines the recommended designs of oncology trials for accelerated approval. Under the draft, sponsors can conduct a single RCT that would support both accelerated approval and verify clinical benefit, or run separate trials aimed at supporting approval and confirming benefit. (RELATED: FDA encourages RCTs in accelerated approval guidance for oncology, Regulatory Focus 26 March 2023)…
