According to a press release from the US Food and Drug Administration (FDA), on March 24, 2023, the agency issued a new draft guidance titled Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics. This guidance is focused on the design of clinical trials in cancer therapies hoping to get evaluated under Accelerated Approval protocol. This approval pathway is often utilized for cancer therapies, as this takes into account the serious, life-threatening character of many cancers. Furthermore, cancers tend to have a lot of intermediate endpoints that can be used effectively to predict potential benefits for patients.
Dr. Richard Pazdur, director of the FDA Oncology Center of Excellence, had this to say about the new guidance:
“Today’s draft guidance provides recommendations to sponsors for designing clinical trials to support accelerated approval. Building quality and efficiency into the design of oncology clinical trials is a crucial component in providing maximum benefit to those living with cancer.”
Some of the specific recommendations mentioned include the design of the trials themselves, methods for improving the available data at the time of Accelerated Approval, and taking concrete steps to reduce uncertainty about the drug’s effect by promptly beginning confirmatory studies. These postmarketing trials are a requirement under Accelerated Approval in order to fully verify the clinical benefit predicted in the earlier trial.
The guidance also discusses the use of a one-trial approach, as in certain cases, separate clinical trials may not be necessary because a longer-term follow-up period in the same trial could help fulfill the requirement of postmarketing confirmation of clinical benefit. The guidance also highlights that confirmatory trials that are already underway once Accelerated Approval occurs are favored to obtain a timely verification of benefit, which will minimize uncertainly for the patient community.
A commentary in the New England Journal of Medicine from 2022 describes the concepts that were incorporated into the guidance. The FDA’s Oncology Center of Excellence also kicked off the initiative Project Confirm, which is intended to push for transparency regarding outcomes in Accelerated Approval for cancer therapies, as well as encourage research and debate focused on the protocol.
