REGULATORY UPDATE: Peter Marks Outlines CBER’s Plans for Near Future

Peter Marks, MD, PhD, director of FDA’s Center for Biologics Evaluation and Research (CBER), stated that in the coming year, the agency is planning new initiatives and resources to aid the blood and biotherapies community, and is investing in ways to accelerate its approval process. Marks made the comments earlier this week on a webinar hosted by Alliance for a Stronger FDA.

CBER is requesting a $10.5 million budget increase for fiscal year 2025. The increased funding is intended to address issues including supply chain integration, IT modernization and restoring funds rescinded post-COVID for vaccine surveillance, as well as to support new initiatives to better address infectious disease transmission and approval for rare disease gene therapies.

Marks said among its many upcoming projects, CBER is planning to develop new guidances to address infectious disease risk. “Our priorities for the current year have included updating our tissue guidance to reduce the risk of infectious disease transmission, as there was a recent outbreak of tuberculosis in tissue grafts, which we need to address,” he said.

Marks said that as gene therapies continue to expand, CBER will devote more resources to its approval and review process for gene therapies. “We are also interested in ensuring people understand how to apply accelerated approval to rare disease gene therapies, an area we are working on,” he noted. He added that the agency is developing a new platform technologies guidance; it will also likely be developing a new platform technology guidance specifically for genome editing.

Additionally, CBER will be working to initiate and execute the Secure Tolerance Alliance Registry (STAR) for enhanced communication regarding rare diseases. The agency will also be expanding collaboration with the European Medicines Agency for streamlined global submissions.