October 29, 2020

The Niche

Knoepfler lab stem cell blog

What is the FDA’s appropriate role in regulating medical innovations such as stem cells?

FDA stem cellsWe need medical innovations, but what is the appropriate way for agencies like the FDA to regulate the process and fight bogus interventions based solely on hype?

Without innovation, medicine becomes stagnant and hope fades for patients who have any one of numerous conditions that are currently inadequately addressed by medicine.

I can relate on a personal level to need for medical innovation as a survivor of a very serious form of prostate cancer. I’m currently in long term remission 4+ years after surgery, but it could come back some day. At this time, medicine has remarkably little to offer patients with recurrent prostate cancer. For example, I would say the prostate cancer field is way behind the breast cancer field. Innovation is desperately needed for recurrent prostate cancer and many other medical conditions.

On the other hand, the arena of medical innovation, whether for cancer or stem cells, is ripe to be exploited by those peddling snake oil. It always has been and probably always will be.

How do we tell the difference between those with good intentions to innovate and the exploiters in the stem cell world? It can be extremely difficult to distinguish between them even for regulators such as the FDA. Of course many entities may fall in the middle in a gray zone.

We need the FDA in the stem cell world, but what is the appropriate role for the FDA in regulating innovative stem cell treatments and products? How does it find the sweet spot of not too much regulation that stifles stem cell innovation and not too little regulation that allows patients and potentially the whole stem cell field to get hurt?

Are FDA programs such as Fast Track, Accelerated or Priority Review, Breakthrough Therapy designation enough to promote innovation while protecting patients at the same time? To my knowledge no cellular therapy has received Breakthrough Therapy status. Will 2014 be the first year we such a positive move?

What about compassionate use? What is the best way for the FDA to handle that? Update: what about the new RMAT program?

I don’t think I have all the answers or anything remotely like that to the questions above, but I do believe these are dilemmas that need more open and frequent discussion in the stem cell field.

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