By Sarah Karlin-Smith, May 7, 2025
The US Food and Drug Administration’s announcement of Vinayak “Vinay” Prasad as the new director of the Center for Biologics Evaluation and Research may signal a major philosophical shift in the data required to approve medical products, particularly cell and gene therapies.
FDA Commissioner Martin Makary announced the pick of Prasad, a hematologist and oncologist, in a 6 May email to staff obtained by Pink Sheet. Prasad most recently worked at the University of California, San Francisco, as a professor of epidemiology and biostatistics.
Prasad will replace Peter Marks who resigned from FDA rather than be fired in late March
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Will Prasad Nix Makary’s ‘Provisional’ Pathway?
Prasad also has a long track record of criticizing the agency for making it too easy to get drugs on the market, raising concerns about the accelerated approval pathway and recent gene therapy approvals, such as Sarepta’s Elevidys (delandistrogene moxeparvovec-rokl).
He has supported randomized controlled studies with hard clinical outcomes even as the FDA, including Marks, embraced more flexible approaches, particularly for rare diseases.
Whether Prasad’s views can align with Makary’s recent proposal for a new ‘plausible mechanism’ pathway for rare disease drugs is unclear.
Prasad’s history, which includes criticism of the FDA and pharma’s close working relationship, worries industry.
His appointment “seems counter,” to Makary’s discussions in recent weeks about reforming the drug approval process, particularly for rare diseases, said Nicole Paulk, CEO and Founder of Siren Biotechnology, which is developing gene therapies for cancer.
“Based on [Prasad’s] books that he’s written and the tweets that he has shared, and podcast that he has been on and all of these various public forums, it would seem to be that his position, kind of broadly regardless of modality is pretty anti-FDA, fairly anti-innovation, fairly anti-accelerated anything,” Paulk told Pink Sheet.
Investor Misunderstanding About CDER, CDER Product Oversight
The news of Prasad’s appointment sent biotech stocks plummeting.
A 6 May analyst note from William Blair suggested investors may be confused about the products regulated by CBER and the Center for Drug Evaluation and Research. The analysts said they do not believe there is a risk to the existing development path for most oncology drugs, which are overseen by CDER.
For cell and gene therapies, “there are clearly outstanding questions and increased uncertainty now as we wait to see whether Dr. Makary or Dr. Prasad will have more impact on the guidelines and regulatory development requirements for these novel therapies, particularly in rare diseases,” the analysts wrote. “Dr. Makary has been vocal since being confirmed as commissioner for more accelerated approval opportunities in the cases of ultrarare diseases or therapies with overwhelming efficacy.”
Prasad’s appointment also likely will add to the negative sentiment in the vaccine space, they added.
A Triumph For Evidence Over Hope?
Many critics of the FDA’s approval standards hope Prasad’s appointment will lead to tougher approval standards.
“Like us, Vinay has led several impactful research projects raising concerns around surrogate markers and their lack of association with meaningful clinical outcomes, particularly in oncology,” said Reshma Ramachandran, co-director of the Yale Collaboration for Regulatory Rigor, Integrity and Transparency. “He also similarly expressed concerns around the previous CBER Director’s decision to override multiple scientific and technical review teams to approve Elevidys despite lack of evidence of its efficacy. Hopefully, this is a sign that in his new role, he will listen to his scientific review staff in making regulatory decisions.”
“I think very highly of him,” said Diana Zuckerman, president of the National Center for Health Research. She suggested that Prasad’s track record suggests someone who would not allow products on the market simply because patients lack any treatment, unlike Marks.
“I understand the desire to be responsive to patients’ needs and desire to have hope for a new treatment, but I think it does patients no favor to give them false hope by approving treatments that end up being extremely expensive and not only ineffective, but they don’t follow through with the required post-marketing testing,” Zuckerman said. “So for years they end up on the market with companies that make a lot of money off of them and patients who don’t benefit at all or are harmed financially, physically or both.”
Ethan Perlstein, CEO Of Perlara, a biotech public benefit corporation developing treatments for rare genetic diseases, said the negative reaction to Prasad comes from “traditional bio” because they dislike a person as outspoken as Prasad who “can’t be controlled in some way or is not beholden to them or to anybody.”
Perlstein said he understands why many in the industry see Prasad as a “chaos agent,” but added that he appreciates Prasad demonstrating that he is “not beholden to anybody except his principles,” and seems willing to evolve.
Has Prasad Changed?
Other medical experts who raised concerns about Prasad’s views on COVID-19 and other vaccine issues in recent years acknowledged that before 2020 they liked many of his stances, such as requiring more rigorous follow up on oncology drugs after accelerated approval.
But they were not confident that Prasad still existed.
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“He started out criticizing the COVID vaccines and I think a lot of it is audience capture,” said David Gorski, a professor of surgery and oncology at Wayne State University and editor of the blog Science Based Medicine. “As he drifted into more contrarian positions, he got more likes, clicks praise, as a brave truth teller.”
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Gorski suggested Prasad is a victim of what he calls “evidence-based medicine fundamentalism,” which is “if it isn’t a randomized placebo-controlled, double-blind clinical trial, it’s crap,” Gorski said.
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