The Trump Administration just quickly gutted the FDA including key leadership. What is going to be the impact of this sudden purge?
Former Commissioner Robert Califf recently said it is the end of the FDA as we’ve known it.
Those are dramatic words, but they seem on the money to me. As a biomedical scientist who also follows the legit biotech sphere and the unproven stem cell clinic industry, I’m most focused on CBER, the biologics branch of the FDA. Many of CBER’s essential people are gone. STAT News has a piece listing all the FDA leadership departures.
In addition to the departure of director Peter Marks, I see other key CBER people are gone. While some of these departures like Marks’ are technically resignations or retirements, much of what is going on reflects people being forced out. After Marks left, others may have chosen to follow his exit too, especially given the tough words in his letter of resignation that mentioned “lies” and more from RFK Jr.
What’s left of CBER now?
The CBER organizational chart is missing so many key names besides Marks. It hasn’t been updated in three weeks so who knows what’s left now.
While Julia Tierney is listed as acting director as of March 31, we now know that Scott Steele is the new acting director.
My sense is that even unproven stem cell clinics aren’t sure what’s coming next. Some may be hopeful that Kennedy will usher in a period of deregulation but it could be more chaos than organized reduction of oversight.
How can CBER function like this and what are the implications for good citizen biotechs?
Why damaging CBER is so bad for biotech: muddy waters, slower decisions
It’s worth noting that the biotech industry is also not reacting positively to these FDA changes. Adam Feuerstein had a piece about how biotech and investors have been viewing the changes including Marks’ ouster. That piece and other views I’ve seen reflect a deep sense of uncertainty. Gloominess.
The biotech industry needs stability at the FDA. Otherwise, how do they know how to approach going for INDs, BLAs, and drug approvals. For any given biotech including those pursuing development of biological drugs how do they plan ahead?
There is a long list of questions, reflecting a level of uncertainty that biotech has got to hate.
People are still trying to get a read on what Steele might do at CBER. Does he have his own philosophy or will he mainly do what the higher ups say? What might RFK Jr. order more generally? How much will Makary, who is also a newcomer, have sway and what are his priorities?
At a more nitty-gritty level, how is the FDA going to evaluate data and products from biotech firms? Do inspections? Who’s left to do such things and what will their orders be? The loss of institutional knowledge at the FDA and CBER specifically seems profound. If a biotech or an academic sponsor of a trial reaches out to CBER, is there anyone there to answer who is an expert? You can’t just wing it.
Add it all up and I expect the FDA will be much slower to evaluate and approve new INDs, BLAs, and drugs from biotech. There’s going to be this long, costly limbo when nothing happens for many products and firms. That is bad for patients and negative for the biotech business.
Ideology & politics over data?
Oddly, other products that are seen as priorities by the new FDA leadership may suddenly get approval with seemingly less data.
Why might that be? Beliefs could guide key decisions over data.
One overriding question is the extent to which ideological and political views may impact how the FDA and CBER engage with biotechs and make decisions.
For instance, if you are a cell therapy firm using pluripotent stem cells (to make neural cells or eye cells, etc.), are you now somehow at a disadvantage? Given Kennedy’s apparent preference for adult stem cells like perinatal cells sold by unproven clinics, will other firms like pluripotent stem cell-focused biotechs run into new issues? Or just sit in limbo?
Whatever your product, if your biotech firm happened to promote DEI, are you now in hot water no matter how good your data are? When it comes to FDA evaluation and decision-making on your trial data and products, could you face bias from the agency? Are you even on some list now for investigation?
Putting it another way, more generally, how much are politicians going to micro-manage CBER decision making based on just their own beliefs and the thinking of those above them?
Even if you are a leader of a firm developing an adult cell therapy, which Kennedy might like, you just don’t know what to expect from the FDA now.
Overall, will we see biotech leaders cozying up to Kennedy?
Clinics unleashed, more chaos overall
On the flip side, if under Kennedy’s watch CBER now lets loose the unproven clinics from proper oversight, that’s just going to make things even worse for the good citizen biotech firms.
More unproven clinic customers are gong to get hurt.
The public may get even more confused about “stem cells” too.
The overall picture is likely to be a dysfunctional mess. The cell therapy arena has been struggling with most firms having seen their stock prices consistently dropping. The new FDA and CBER could even be the end for some vulnerable biotechs.