In a recent discussion, Peter Marks, the director of the FDA’s Center for Biologics Evaluation and Research, and Janet Woodcock, former FDA deputy commissioner, highlighted the critical need for regulatory flexibility in developing rare disease therapies. They also emphasized the importance of enhancing gene therapy manufacturing capabilities.


Balancing Regulation and Flexibility

Marks and Woodcock addressed the challenge the FDA faces in balancing strict regulatory adherence with the flexibility needed for rare disease drug approvals. Woodcock noted the internal conflict within the agency, where officials are required to enforce regulations while also being encouraged to exercise flexibility.

“What we’ve seen is a lot of cognitive dissonance internally,” Woodcock said. “People are being asked to, on one hand, enforce the standard, on the other hand be flexible. You can be flexible, flexible, flexible, and then pretty soon, you’re going to break.”

Marks concurred, pointing out that while the FDA has the capacity for regulatory flexibility, the agency’s culture has traditionally favored strict regulation adherence.

“We have people that are smart enough that we can focus on the patient and actually still stay on the right side of the regs, and I think it’s just a matter of, in some cases, just changing how we think about things and how we approach them,” he said.


Accelerated Approvals and Insurance Challenges

Marks, a proponent of accelerated approvals for rare disease therapies, warned of potential complications with insurance coverage for these expensive treatments. He cited the example of Biogen’s Alzheimer’s drug Aduhelm, which received accelerated approval in 2021 but faced significant sales challenges when the Centers for Medicare & Medicaid Services (CMS) refused coverage.

“We may see with Medicaid and gene therapies for sickle cell disease just to call that out, I’m kind of worried about how that’s going to roll out,” he said. “But you can imagine it happening on a broader scale if we change a standard.”


Investing in Gene Therapy Manufacturing

Both Marks and Woodcock emphasized the necessity of investing in gene therapy manufacturing to reduce costs and improve accessibility. Woodcock has long advocated for continuous manufacturing processes to streamline production and lower expenses.

“This would be something that the US could invest in,” Woodcock said. “I’ve been trying for continuous manufacturing for the last 20 years.”

The discussion underscores the FDA’s ongoing efforts to strike a balance between maintaining regulatory standards and fostering innovation in rare disease treatments, particularly in the rapidly evolving field of gene therapy.

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