We 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.
Even with all this wealth of information and research on stem cells, we do not have all the answers to predict either the mechanism or the mode of action with great detail. The reason being that cells are living and are constantly changing, thereby throwing into the mix another variability. The FDA sees this unpredictable to be the main problem even if it were effective in a majority of the population. I guess we and the FDA will learn a lot from clinical trials performed in other countries over time and come to a consensus to frame lesser stringent laws for stem cell application.
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Regulation must take into account the nature of the therapy and the type of business model that it can conform to. There is a huge difference between developing a mass-produced drug and an autologous stem cell therapy. The autologous therapy becomes a non-starter if it is forced into a business model and regulatory methodology that is designed for a mass-produced drug.
I’d say the main game is the regulation of the regulators. Presently regulators are punished if they allow a treatment that is harmful but they get a free pass to stymie a treatment that if it were allowed to progress might be found to be beneficial.
Ultimately, it’s a matter of managing risks: the risk of not allowing a benefit and the risk of allowing harm. In either case, getting it wrong does harm. In every case, a one-size-fits-all approach fails.
I believe he answer lies in with the steps being taken by Japan in recent legislation. They see the potential that regenerative medicine can play, to not only cure incurable diseases, but to have a huge positive impact on healthcare costs in an aging population. The road they are taking is a rather simple two step approach. 1) Demonstrate safety, with signs of efficacy, to obtain provisional approval that would allow a therapy to reach patients immediately. 2)Use the investigational data collected through this provisional approval to demonstrate efficacy that would make provisional approval permanent.