There is considerable interest in FDA regulatory reform here in the U.S. for stem cell therapies. The goal is to get new treatments to patients faster, but is there a way to do that without sacrificing proven safety and efficacy?
You need convincing data on both safety and efficacy or speed becomes dangerous.
A group called the Bipartisan Policy Center (BPC) recently issued a report related to these central questions of American regulation of new stem cell products. You can see a screenshot from the cover of the report above. The report argues for a new paradigm of FDA regulation of stem cells with reduced regulatory steps.
While I support the notion that there need to be reforms at the FDA related to regulation of stem cell-based therapies, looking at it scientifically based on the current state of stem cells in the US, the potential cons of what the BPC report recommends outweigh the possible pros.
Pursuing the BPC plan at this time would be a high-risk experiment that could yield benefits and harms or most likely some complicated combination of both, but again I believe the risks are considerable making the report imprudent overall. Others, including some stem cell colleagues feel otherwise so I think a lively debate could prove to be healthy on this topic.
Below I outline the potential pros and cons as I see them.
Possible Pros. The report does a good job of highlighting the potential positives that could come from the proposed regulatory reforms:
- Speeding therapy to patients. The most prominent possible upside would be speed in the form of getting new stem cell therapies into patients more quickly.
- Helping American competitiveness. A second positive, depending on one’s perspective, would be possibly increasing American competitiveness in the global stem cell arena.
- Generating money. Finally, because the plan specifically allows companies and non-profits to charge for experimental stem cell therapies, those entities doing the research could benefit from that income and in theory that could support furthering of the research.
Possible Cons. Perhaps because the report is intended to convince readers of its thesis that we need regulatory reforms that accelerate getting stem cell therapies into patients, it doesn’t discuss risks or potential cons to the recommendations. However, as a scientist I find it disappointing that the report ignores potential risks to its recommendations.
In science, especially clinically-related science, we cannot afford to ignore risks. For this reason, the report reads as being somewhat more political than scientific, perhaps not an entire surprise given that two former politicians led the way on it.
The significant cons to the recommendations if implemented include the following:
- Risk of medical harm. When the BPC Plan on Stem Cells characterizes stem cells as different than drugs, it goes against decades of science. Biological drug products released onto the for-profit market without full vetting will pose some risks of increased medical harm to consumers. For instance, dropping the requirement for Phase III trials as they recommend in some cases would be risky. Ignoring that increased risk seems unwise as it needs to be part of the equation and discussion in risk-benefit analysis for any new regulatory paradigm.
- Charging research subjects. There is a tradition in biomedical science of not charging patients for receiving experimental treatments. In fact, more often patients are paid to be subjects. I believe that taking patients’ money to allow them access to an unproven, experimental treatment is unethical. Unfortunately, some are already doing this in the U.S., but the BPC report if implemented would make that practice far more common. I understand that the translational stem cell field could benefit from generate some funds in support of clinical studies, but this shouldn’t come from patients.
- Unintentionally helping predatory clinics. The BPC recommendations, while still requiring some approvals such as an IND, are significantly weaker than the current regulatory framework. From what I’ve heard from the for-profit clinic folks, they see this report, even if it isn’t implemented and even though it still requires an IND, as a victory. Why? Because they believe that the report represents a shift in their direction ideologically. They see it as somewhat of a capitulation by the academic and biotech sectors toward the notion that the FDA is wrong on stem cells. This is very worrisome. The report even uses some of the memes of the stem cell clinics and their proponents such as arguing, as mentioned earlier, that stem cells are not drugs and that patients have a right to get transplants of their own cells even if those cells have been turned into a product.
- Encouraging global deregulation. This plan could also be taken as a template in many other countries around the world for their own regulatory changes toward weaker oversight, which could amplify risk to thousands more patients globally.
- Fostering a sense of potentially unhealthy, international competition. I’m an American and we have a great country here, but I have reservations about framing stem cell regulatory debates in a nationalistic or political kind of way that could lead to harmful competition. Again to me the BPC report comes off as too political and not scientific enough, and that is especially true when it discusses national priorities. There is a palpable sense in the report that those who wrote it are worried about the US falling behind Japan on stem cells.
Overall, I recommend that the FDA not adopt the BPC recommendations because that would be just too risky and raises serious bioethical concerns. We need a different plan that more cautiously approaches regulatory changes and openly acknowledges that there would be risks.
One such plan I could potentially support would be focused squarely on a relatively simpler path to FDA triggering of Fast Track, Accelerated or Priority Review or Breakthrough Therapy Designation for stem cells.