The FDA’s top biologics chief and a former deputy commissioner on Tuesday discussed the need for regulatory flexibility for rare disease therapies and underscored the need to bolster gene therapy manufacturing.
Peter Marks, the FDA’s biologics center director, and Janet Woodcock, former FDA deputy commissioner who retired earlier this year, discussed how the agency should be able to balance following regulations with exercising some flexibility for rare diseases.
“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 added that he thinks the FDA already can leverage a fair amount of regulatory flexibility but that the culture of the agency has historically prioritized strict adherence to the regulations.
“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.
Marks, who’s been pushing for more accelerated approvals for rare disease therapies since at least August 2023, warned that changing the current framework could also exacerbate issues with insurance companies hesitating to cover the expensive new therapies, saying that that has already been an issue with some accelerated approvals. Biogen’s former Alzheimer’s drug Aduhelm won an accelerated approval in 2021 but failed to muster any sales as CMS declined to cover it, and eventually Biogen sold it.
“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.”
Marks and Woodcock both emphasized that investing in manufacturing for gene therapies is key to bringing down their cost.
“This would be something that the US could invest in,” Woodcock said. “I’ve been trying for continuous manufacturing for the last 20 years.”