Do You Think That Biopharmaceutical Companies Receiving An FDA Accelerated Approval Should Be Required To Enroll Patients In A Confirmatory Study Prior To Launch?

THE PURPOSE OF THE FDA’S ACCELERATED APPROVAL PROGRAM is to expedite the availability of new treatments for patients with serious conditions and to fill an unmet need. Any delay in making the treatment available effectively negates the concept of acceleration. However, until clinical benefit is confirmed via a properly designed and conducted confirmatory trial, there should be some oversight of the product’s use, and experience data should be gathered to support the continuation of availability until results of the confirmatory trial are known. If patients who were prescribed a drug made available by Accelerated Approval were required to participate in a “registry” type study, including the submission of outcomes data, the drug sponsor and the FDA would be able to monitor “real world” safety and efficacy. This registry data would be unsuitable for converting an Accelerated Approval to full approval, but it could help to identify issues for further study. Additionally, physicians who prescribed Accelerated Approval drugs for patients and contributed to the registry would be more likely to be engaged clinical trial investigators.

MARY ROSE KELLER is VP, head of clinical operations at Acrivon Therapeutics. She has 30+ years of industry experience.