New FDA Draft Guidance: Considerations to Support Accelerated Approval of Oncology Therapeutics

 This draft guidance covers both drugs and biologics and provides recommendations for designing clinical trials that support approval. It notes that single-arm trial desings and response endpoint are most common for oncology accelerated approvals, and then list 5 limitations of single-arm trials.  It goes on to suggest that "properly designed and executed, a randomized controlled trial can address the limitations of single-arm trials, including but not limited to, the following way" and then lists the 5 ways. A sixth value for a randomized trial is then discussed:

Another potential advantage to conducting a randomized controlled trial to support accelerated approval is that, in appropriate cases, longer term follow-up in the same trial could fulfill a postmarketing requirement to verify clinical benefit. This “one-trial” approach maintains efficiency in drug development and can provide early access to a drug using the accelerated approval pathway, while ensuring that a postmarketing trial is fully accrued and well underway to verify longer term benefit in a timely fashion.

 The draft guidance then goes onto to provide recommendations for both randomized and single-arm trials before concluding with a section on a 'Confirmatory Trial Following Accelerated Approval".