CBER chief says pilot program will justify increasing reviewer headcount

Cell and gene therapy manufacturers may not like the idea of spending more money on US Food and Drug Administration (FDA) reviewers, but a top official said if a new pilot project goes according to plan, it will justify hiring more reviewers to help speed new treatments to market.

Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER), outlined the key challenges faced by his center and how he is working to address them during a webinar hosted by the Alliance for a Stronger FDA on 4 December. In particular, he noted that there are challenges with manufacturing processes, clinical development timelines and achieving global harmonization across regulatory regimes.

In September 2023, CBER launched the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program to address these challenges. The program allows sponsors to get advice and request ad hoc communications from reviewers on product-specific development issues, including clinical study design, choice of control group and clinical trial patient selection (RELATED: Convergence: CBER chief discusses priorities, touts gene therapy approvals, Regulatory Focus 5 October 2023)

Marks said he was impressed by what CBER was able to achieve with Operation Warp Speed in developing COVID-19 vaccines collaboratively with manufacturers. He said the agency will be using lessons learned from that as a basis for the pilot to help bring new cell and gene therapies to market without sacrificing quality, safety or effectiveness.

“It will require additional project managers, it will require additional reviewer time, and my guess is the staffing up that will need to happen, if we were really to expand this significantly, would require us to get dozens of additional staff members,” Marks said.

The CBER director acknowledged that some in the industry may be hesitant about paying significantly more to increase the center’s reviewer headcount. However, he’s also confident that when they run the numbers, they will conclude that potentially adding up to 40 more reviewers could make business sense, if it means they can get their products to market sooner.

“For me, the beauty of this is we can get things to patients that much more rapidly,” Marks said. “I do believe that Congress, and those who pay the bills, have the right to see us demonstrate that this really will work. And that’s the reason for doing the pilot, so that we can get some data to show them that it will make a difference.”

Marks also noted that, more broadly, the FDA recognized that the gene and cell therapy sector has seen massive growth, which led to a reorganization at CBER to create the Office of Tissues and Advanced Therapies (OTAT) super office (RELATED: FDA elevates OTAT to “Super Office” within CBER, Regulatory Focus 30 September 2022).

“We really have wanted to increase our interactions with various equity holders, including industry and patient advocacy groups, and this will allow us to do so,” Marks said. “We very much want to improve the timeliness and consistency of the feedback that we give, and this will help us, and it allows us also to launch our initiative to really focus on what we can do to get needed therapies to patients.”

CBER chief says pilot program will justify increasing reviewer headcount

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