Vinay Prasad has only less than two weeks left at the FDA atop CBER. I’ve been trying to imagine where CBER goes from here after he exits. It feels like FDA Commissioner Marty Makary and RFK Jr. will likely be the real leaders of CBER moving forward on key decisions. Under that pressure, will the new CBER director just be a “yes” person? Probably. Some thoughts: Landmines await Vinay Prasad’s successor at the FDA, STAT.
Speaking of the FDA, former Commissioner Scott Gottlieb is enthusiastic about the new framework. We’ll start there.
Gottlieb on more flexible biologics oversight at the FDA
FDA’s Promising New Framework for Rare Genetic Diseases, JAMA Health Forum. This is by Gottlieb and Maarika Kimbrell.
The emerging flexible framework (which arguably started with Peter Marks a few years back) can make genetic therapies for rare diseases a quicker reality, which seems great.
It comes with big risks though. Much will depend on how it is implemented moving forward.
The evolving flexibility is based on plausible mechanisms.
Also, it’s not clear that plausible mechanisms-based approvals will be limited to bespoke gene therapies. In other contexts, such as for cell therapies (also mentioned in draft guidance), utilizing such a regulatory mechanism could be even riskier. Why? Cell therapies may have a tougher time invoking plausible mechanisms unless they are combination cell-gene therapies.
The article concludes “The FDA’s new draft policy aims to create a pathway in which personalized genetic therapies can advance with far less friction from the laboratory to the clinic and, ultimately, the market. The efficiency of this framework depends on the FDA continuing to refine its requirements for platform approval by broadening the established scope of permissible personalization. These steps will build on the agency’s recent efforts to enable more children to benefit from the molecular tools now within reach.”
The risk-benefit ratio with these flexibilities will also depend on the types of conditions that are eligible. How limited will this be to N=1/bespoke-type conditions? How strongly will politics sway things here?
More recommended reads
- Sammy Hagar claims he undergoes stem cell treatment to keep him in performing shape: “A singer cannot get trashed and still pull off shows”, Guitar.com. He reportedly gets IVs of “young” stem cells twice a year. This sounds like perinatal cells. Ozzy did this kind of dubious stem cell stuff too.
- This method to reverse cellular ageing is about to be tested in humans, Nature. I’ve written before about how this OSK in the eye trial from David Sinclair seems so risky. The preclinical mouse studies are interesting, but it’s not entirely clear to me that it’s time yet to be doing a trial in humans. Hopefully the FDA reviewers who cleared the IND saw some more impressive data than is in the public domain.
- Federal Agency Unveils Three Potential Osteoarthritis Treatments, NYT. These are interesting projects, but not necessarily more so than other regenerative efforts in this important area. The size of these federal grants is astonishing with just one of them at $42M. Think about the fact that the feds could fund 20 R01s with the amount of funding for just one of these ARPA-H grants. I don’t know what you think. Is it worth it? To me that massive funding sets the bar super high. These three better do something amazing.
- Our SCOPE stem cell outreach project has a stem cell white paper in dozens of language, but not yet Finnish. If you speak Finnish would you be up for helping? Please let me know. Good for your CV.