How might Trump’s nominee to lead the FDA, Martin Makary, change the agency? Should we be worried or hopeful?
I’ve been closely following the FDA and its commissioners ever since I started The Niche in 2010. I’ve been particularly interested in how these leaders impacted FDA actions on cell and gene therapies. How might Makaray impact this overall area of regenerative medicine? A central problem in our arena is that hundreds of clinics are selling unproven and non-FDA approved cellular drugs.
Makary is very different than past commissioners so it’s important to think about how things might play out if he becomes FDA Commissioner, which is almost certain to happen.
Makary’s background
Dr. Makary seems like a serious, successful biomedical scientist. He has a vast track record of strong publications as a cancer surgeon and professor at Johns Hopkins. You don’t get to that point by regularly accepting baloney about health or science.
By contrast, when we think about the many hundreds of unproven stem cell clinics across the U.S., most of them are selling highly questionable offerings without any good data. In some of the worst cases, it is junk in a syringe.
I don’t see Makary being a fan of that even if more broadly he has been critical of the FDA at times.
Revisiting Commissioner Scott Gottlieb’s tenure as FDA Commissioner
For context, let’s look back at the FDA during Trump’s first term. In early 2017 he nominated Scott Gottlieb to be FDA Commissioner.
At that point, I was worried about what Gottlieb might do at the helm of the FDA.
However, Gottlieb was one of the better FDA leaders when it came to starting to tackle unproven stem cell clinics. He was outspoken and active on this front.
I found it a pleasant surprise in part because he had written previously about the FDA over-regulating stem cells. Early on I wondered if he might radically change the FDA or weaken it.
He didn’t.
When Gottlieb became Commissioner, I’m betting it became clear for him just how much of a problem these unproven clinics were.
Martin Makary and the FDA
If I had to predict, I think the same thing will happen with Makary. Most likely he’ll be inclined to take the clinic problem seriously.
There are some reasons to be concerned too though.
The first is having RFK Jr. potentially above Makary as HHS Secretary. As I’ve written Kennedy has falsely claimed that the FDA is suppressing stem cells. I imagine he means unproven stem cell clinic offerings.
Another concern is that Marty Makary took some risky positions during the heart of the COVID pandemic. For example, he seemed to embrace the herd immunity idea and downplay the importance of some of the later COVID vaccines. More broadly he seems supportive of vaccinations though, unlike Kennedy.
Kennedy and Makary could end up in conflict
If Kennedy is HHS Secretary and he pressures Makary as head of the FDA to stop taking enforcement action on unproven cell therapy clinics, what will happen?
Makary seems unlikely to be pushed around by Kennedy, but what if Trump applies pressure on this front too?
I don’t know that Trump cares about stem cells but it’s hard to predict what might unfold. During Trump’s first term, some on the right embraced unproven stem cells.
So far in 2024 the FDA and its biologics branch, CBER, have done far more on unproven clinics than in any past year. Could that momentum be lost?
I also frankly find it somewhat worrisome that Makary is such a fixture on Fox News. We need a non-partisan FDA commissioner. Most of the time with past commissioners, once they took the reins they seemed less political. That could happen here too.
On a quick look, I don’t see that Makary has written or spoken specifically about stem cells so that does make things harder to predict about what he might do in our arena.
Envisioning a Makary FDA
Overall, hopefully Makary will stand up for science-based medicine and effective FDA oversight, including of cell and gene therapies.
The broader regenerative medicine field is at an exciting point and needs a robust, logical FDA as a partner. That requires a thoughtful commissioner who focuses on data. One scenario is that Makary maintains the focus on data-driven decisions, but finds creative ways to speed up biologics evaluation and perhaps approvals. That could be a win-win.
On the other hand, I can see Marty Makary finding himself in tough spots at the FDA where his instincts as a physician and scientist may point one way, while politics point the other.
Which will win out?
A Rahman-
You seem to be suggesting that medicines be approved by popular vote, not by evidence: you said:
“Yes, data are essential, but regenerative medicine policy cannot be data-driven alone. That’s the kind of myopic technocracy that causes people to suffer. Public policy of any kind – stem cell or otherwise – needs to be HUMAN-driven first and foremost. We can’t allow statistics to make us deaf to the voice of the people”.
I would argue that people suffer not when the FDA requires scientific evidence of safety and efficacy, but when predatory clinics sell their products through – often made up – patient testimonials. People suffer because of the charges – $10,000 or more for whatever the clinic wants to administer – and because of the real harm that we know that people have suffered because of lack of safety regulation…or any regulation at all.
My mother, and probably yours, asked me if I would jump off a cliff because all of my friends were jumping off the cliff. Don’t you agree with my mother that the answer should be no?
I suggested nothing of the sort. I actually stated that data are ESSENTIAL, but data ALONE – especially when the focus is only on certain kinds of data – are insufficient. My Ph.D. is in Politics, and in American politics the voice of the people is ALSO essential in policy-making. This is called democracy.
The availability and affordability of SCT is not and cannot be a strictly scientific/academic pursuit. The FDA and all other agencies exist within an inter-connected socio-political ecosystem, one whose regulation is based on the balancing of interests.
Of course, safety is part of this balancing equation. So is efficacy. However, the need for new avenues by which unique medical modalities such as SCT can be made available to meet the crushing epidemic of chronic illness is likewise a variable in the equation.
Thanks for bringing this up. Many of us are wondering about this.
Trump has been consistently critical of both the FDA bureaucracy and the pharmaceutical industry, specifically as it concerns the lack of approval of novel medical solutions to chronic disease. His push for the Right to Try Act is indicative here.
And if we are to take Kennedy’s posts on X seriously, he is even more ardent than Trump on the issue, specifically mentioning SCT as part of his agenda. So, as leader of HHS I believe he will do what he can to make SCT more widely available and affordable.
Makary’s position on SCT is more difficult to discern. To my knowledge, he’s never specifically addressed the issue. Time will tell. I believe he is open to doing things differently and has no problem breaking from established dogma or convention to move medical innovation forward.
All in all, the current FDA has effectively been hands off when it comes to the proliferation of SCT clinics. Sure, there have been a few warning letters and lawsuits, but neither the growth in the number of clinics nor the American people’s desire for SCT has abated in the least.
Yes, data are essential, but regenerative medicine policy cannot be data-driven alone. That’s the kind of myopic technocracy that causes people to suffer. Public policy of any kind – stem cell or otherwise – needs to be HUMAN-driven first and foremost. We can’t allow statistics to make us deaf to the voice of the people.
Furthermore, SCT policy must be EVER-CHANGING to meet the needs of the people. Doing things the same way just because that’s the way it’s always been done is backward, dangerous, anti-scholastic. The current FDA is working with certain clinics and that’s great. But we need much, much more.
Thanks.