New FDA Commissioner Dr. Scott Gottlieb, M.D., has in the past touched on stem cells and regenerative medicine therapies in speeches or written comments prior to starting his tenure at the agency. Now that he is Commissioner, he is poised to have direct impact on our field rather quickly and potentially with major changes in store.
How might that unfold?
In one of his first statements that includes stem cells and regenerative medicine as Commissioner, Gottlieb perhaps gave us some hints within a new blog post on the FDA site entitled, “How FDA Plans to Help Consumers Capitalize on Advances in Science”.
Here is the part of his post related to stem cells and regenerative medicine:
“Our Center for Biologics Evaluation and Research (CBER) is implementing the Regenerative Medicine Advanced Therapy, or RMAT designation. This new process provides another pathway to access FDA’s existing expedited programs, and is available for certain cell therapies, therapeutic tissue engineering products, and certain combination products. The goal of these efforts is to help foster the development and approval of these novel products. We’ve already received almost two dozen requests for RMAT designation and granted four such designations to date. To continue to advance these opportunities, we’ll be announcing this September a comprehensive framework for the development and proper FDA oversight of regenerative medicine. This new policy effort will comprise a series of new guidance documents covering many aspects of the regulation of regenerative medicine products. It will be announced as part of our Innovation Initiative. It will delineate our policies for appropriate and efficient regulatory oversight of regenerative medicine products, in order to demonstrate their safety and effectiveness. It will also create an accessible framework that will enable providers to more easily collaborate on proving these principles for regenerative products that are advanced within local medical institutions. We want to help facilitate these scientific advances, which hold out tremendous potential for treating and even curing diseases. To achieve these goals, we need to make sure that we have a modern regulatory framework in place that can allow innovators to meet the statutory requirements for demonstrating safety and effectiveness.”
Can one read any tea leaves in there?
While we will have to wait until September to get more clarity, this text has some interesting tidbits. The first thing that jumps out at me is the rapid timeline in terms of CBER having new, hopefully clear frameworks by September (that’s only 2 months from now) on specific types of regenerative medicine. I see that as a positive. Second, the concept of an “accessible framework” suggests perhaps the FDA will include guidances (finalized or draft at that time?) that are clear to the public and to a diverse group of stakeholders, and hopefully include concrete examples of various scenarios. For instance, are fat stem cell products like SVF by definition biological drugs? If sometimes not, will that depend on their homologous use? What about non-homologous use of bone marrow products? Where do the amniotic products increasingly popular with clinics fit in here? I could go on probably with 20 questions.
Finally, the last sentence of the quote above has the verb “allow” as pertains to regenerative medicine practitioners, which struck me. The use of that verb suggests a smoother path at the FDA for accelerating investigative stem cell therapies perhaps also with a lower initial hurdle for entry into the FDA review process for investigators and their products. The stakes are big here. I’m very curious to see if the FDA’s new framework on regenerative medicine will hit a sweet spot of promoting innovation while not lowering oversight standards. That’s not an easy task. Whatever the framework, it’d be refreshing if CBER actively enforces its policies on stem cells and regenerative medicine in a prompt, consistent, and clear manner.