Vinay Prasad at CBER: What His Appointment Signals for the Future of Biotech Regulation
Potential implications of the Prasad appointment and some early evidence that maybe it's not all doom-and-gloom? I guess we'll find out soon enough. Also an addendum on Rev Med's KRAS G12D inh data.
The FDA has a new director for the Center for Biologics Evaluation and Research (CBER), and it’s someone who has spent a good chunk of his public career questioning how the agency does its job. Dr. Vinay Prasad—hematologist-oncologist, UCSF professor, podcaster, and author—has been appointed to lead the division responsible for regulating vaccines, gene therapies, cell therapies, and other advanced biologics.
It’s a bold pick. And depending on your vantage point in the drug development ecosystem, it’s either a refreshing push for rigor or a potential headwind to innovation. The reality is likely somewhere in between. But one thing is clear: this appointment is going to shape the tone and tempo of biologics regulation for years to come.
Who He Is—and What He Stands For
Dr. Prasad is best known for his criticism of the FDA’s accelerated approval pathway and the increasing use of surrogate endpoints—things like PFS, ORR, or MRD-negativity that don’t always translate cleanly to improvements in survival or quality of life. He’s argued that the bar for approval should be higher, with randomized controlled trials (RCTs) and hard outcomes like overall survival as the gold standard.
There’s an underlying principle here that’s hard to argue with: medicines should work, and we should be confident they do before giving them to patients. But the challenge lies in how this philosophy gets applied in the context of CBER, where trial designs often have to bend to the constraints of biology, logistics, and ethical tradeoffs. Prasad’s views, if enforced too rigidly, could put some important programs in a tough spot.
Why This Matters for the Sector
CBER regulates some of the most complex, high-risk, high-reward platforms in biotech. We’re talking about cell therapies, gene therapies, and vaccines—areas that already face scientific, operational, and commercial hurdles. Add regulatory uncertainty, and you’ve got an even steeper hill to climb.
An example to ground us:
Arcellx and Gilead’s anito-cel, a BCMA CAR-T therapy for multiple myeloma, is seen by some clinicians as a potential (and maybe better) alternative to cilta-cel. Its appeal isn’t necessarily superior efficacy— but a cleaner safety profile, fewer prolonged cytopenias, long-term neurological AEs, and potentially less neurotoxicity. By most accounts, that is a clear differentiator.
But under a strict “first drug gets the accelerated approval, everyone else must show superiority” framework, which Prasad has indicated in his past public comments (you can think of this as an “anti me-too” stance), anito-cel would maybe need to run a head to head study against cilta-cel? And head-to-head trials in autologous cell therapy are, at best, logistically challenging and cost-prohibitive. Why would JNJ/Legend, the makers of cilta-cel, manufacture product for a study like that? A study that could potentially show that their own blockbuster product is inferior to a competitors.
In oncology and rare diseases, competition between drugs in the same mechanistic class expands access and gives physicians options. Some patients tolerate one drug better than another. Some clinics are equipped to deliver one therapy more easily. While having a definitive answer to which BCMA CAR-T is better in a head-to-head would be scientifically interesting, the reality is that it isn’t practical and may limit therapeutic options for sick patients.
Prasad’s past comments that only the first drug in a class should be eligible for accelerated approval feels both anti-competitive and anti-patient. It also may help lock-in mini monopolies by freezing out innovation. And you’re sending a message to the capital markets that there’s no point in developing a safer, more scalable version of an already approved product.
A Closer Look at Prasad’s Skepticism
Dr. Prasad has been a leading voice in raising concerns about the overuse of surrogate endpoints. And to be fair, there are examples where accelerated approvals didn’t pan out—confirmatory trials failed, and some products were ultimately withdrawn. His call for a tighter link between early data and long-term outcomes isn’t unreasonable.
But the system has already been moving in that direction. Today, sponsors are expected to have confirmatory trials up and running at the time of approval, with clearer expectations for when results are due. In other words, some of what Prasad is pushing for is already becoming policy. The risk is that we overcorrect, and start applying one-size-fits-all rules in settings where nuance is essential. Perhaps the previous regime already moving in Prasad’s ideological direction is enough and no further stringency will be applied, but we’ll just have to wait and see.
The point about surrogate endpoints looms large and is particularly concerning for Arcellx and Gilead. The anito-cel example is even more dire when you dig into it a bit more. The companies are hoping to file for approval based on the single-arm iMMagine-1 study in 4L+ MM – that path looks to have more questions now that Prasad is at the helm of CBER. Moreover, the confirmatory iMMagine-3 study (2-4L MM) has MRD-negativity as one of its primary endpoints, the idea being that this could enable an earlier path to approval as survival data accumulates. How does that square with a CBER head who has been broadly critical of surrogate endpoints and specifically critical of the value of MRD in MM?
Vaccines and Rare Disease: More Watchpoints
Prasad’s views on vaccines are well-documented. He’s voiced concern over COVID-19 vaccine mandates, particularly for younger populations, and questioned the use of mRNA platforms without robust RCTs. That perspective may result in longer timelines and stricter data requirements for next-gen vaccines.
On the rare disease side, he’s been critical of how orphan drug incentives are used and how patient advocacy can sometimes blur the line between stakeholder and sponsor. These are valid points to consider, but again, the question is how they get operationalized. Many rare disease programs simply can’t be tested the same way as large oncology indications. Flexibility isn’t about being permissive—it’s about being realistic.
Interested in becoming a BPS sponsor?
A sponsored post is 100% accessible to all subscriber levels and gets your brand, product, or service in front of my audience of BioPharma Industry decision-makers right at the beginning of my next post.
If you are interested in featuring your company, product, or services in my next post as a Big Pharma Sharma sponsor, please reach out at info@bigpharmasharma.com. Click the button below to learn how you can sponsor the next edition of Big Pharma Sharma.
The RCT Question: Where Philosophy Meets Pragmatism
At the heart of all this is Prasad’s strong preference for randomized, survival-driven trials. That’s the gold standard – I don’t think anyone would disagree with this. But not every disease allows for that and not every patient can wait for a definitive OS readout.
Real-world drug development involves tradeoffs. A good-enough surrogate backed by solid biology and early efficacy can sometimes justify early access—especially when confirmatory trials are already underway. The accelerated approval pathway was designed for this exact situation. I would agree the “confirmatory” portion of this was not always enforced, but as of late that hasn’t been the case. Companies are now having to think about whether it is financially and strategically more optimal to enter directly into a randomized P3 study or go the single-arm P2 + confirmatory P3 path. The concern isn’t with demanding better data—it’s with applying that demand in ways that ignore clinical urgency, practical feasibility, or meaningful differentiation.