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CBER chief floats rare disease hub to better communicate across centers

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SAN DIEGO – The FDA’s biologics center director Peter Marks said the agency is considering the creation of a rare disease hub to better combine the expertise of both its drug and biologics experts.

Speaking during a panel at the Drug Information Association’s annual meeting, Marks made a pitch for the need for a rare disease hub shared between both CBER, the biologics center, and CDER, the drugs center. He said unlike a center of excellence, it would be housed in a single center, and allow the two centers to combine their expertise.

“If we were to take all of these and put them in a center, it would actually be counterproductive,” he said.

He added that rare disease products make up about half of applications reviewed by CDER and about 60% of those reviewed by CBER.

Comparing the hub to the Oncology Center of Excellence, Marks said he’d like to take a lesson from the OCE’s communication style, which makes it easy to get answers from a single point of contact, according to oncology drug sponsors.

Marks also responded to a question about the reauthorization of the rare pediatric disease voucher program, which could sunset this fall if lawmakers don’t reauthorize it.

“We know how important it is, but it’s beyond our control, and as I’ve been told by my head of legislation, we do not lobby Congress,” he said with a chuckle.

Responding to a question about the START pilot program for rare disease, which mirrors the Operation Warp Speed model, Marks said the agency would need to significantly staff up if it becomes a permanent program. He hinted that those resources could potentially be a part of PDUFA VIII negotiations in the next few years.

“The only way it’s going to work without me having to get really big bags under my eyes, is if we hire a whole lot more people and make the same focus in CDER because it is labor-intensive,” he said.


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