To Improve Accelerated Approval Pathway, Look Abroad, FDA Officials Say

Director of the U.S. Food and Drug Administration’s (FDA’s) Oncology Center of Excellence Richard Pazdur, MD, has suggested that the agency’s accelerated approval pathway could be improved by following the example of international regulators’ expedited approval processes.

The FDA accelerated approval pathway has faced criticism for not requiring drug companies to confirm clinical benefit through follow-up studies in a timely manner. As Congress and the agency seek to reform the process, expiration dates could incentivize manufacturers to produce these data. According to Dr. Pazdur, “an ongoing confirmatory trial at the time of accelerated approval was associated with a shorter median time to verification of benefit.”

Overseas, most expedited approvals rely on endpoints that are likely to predict clinical benefit upfront, with fewer E.U. approvals based on single-arm clinical trial findings compared to the U.S. approvals.

“Another notable feature of programs in the E.U., Switzerland, and U.K. is that only new molecular entities are considered for expedited approval. This contrasts with the U.S. and others, which may also grant expedited approval for supplemental applications,” Dr. Pazdur, Director of the Division of Hematologic Malignancies in the FDA’s Center for Drug Evaluation and Research, and coauthors wrote in a recent JAMA Oncology opinion piece. “Additionally, unlike accelerated approval in the U.S., the expedited approvals granted by the E.U., U.K., Australia, and Switzerland are time-limited and expire. In the E.U. and U.K., [conditional marketing authorization] expires yearly and must be renewed with an application and interim report on outstanding obligations submitted at least 6 months before expiration.”

Source: Endpoints News, January 21, 2022.