New CBER Director Prasad a Critic of Accelerated Approvals

After an extended period of unwelcoming capital markets, a new administration has brought cuts to federally funded scientific research and research universities, threats of tariffs on pharmaceuticals, and an executive order from President Donald Trump that calls on the Department of Health and Human Services to take steps to reduce drug prices for American patients.

Not the type of news flow that gets biopharma stocks rallying.

The latest wallop came yesterday when U.S. Food and Drug Administration Commissioner Martin Makary welcomed hematologist-oncologist Vinay Prasad as the new director of the agency’s Center for Biologics Evaluation and Research. Prasad, a professor in the Department of Epidemiology and Biostatistics at the University of California, San Francisco, replaces Peter Marks, who resigned in April. Marks said he found himself at odds with U.S. Health and Human Services Secretary Robert Kennedy Jr. on vaccine policy and criticized his new boss for spreading misinformation about the safety and efficacy of vaccines. Prasad will be responsible for overseeing the division that regulates biologics, including vaccines, cell therapies, and gene therapies.

A prolific writer who enjoys provocative social media posts, Prasad has been an outspoken critic of the agency’s use of accelerated approval and its willingness to approve drugs, particularly new cancer medicines, with little proof of efficacy. In a peer-reviewed setting, he doesn’t foam at the mouth but offers hard-hitting criticism you’d expect from an academic.

“Drug regulators’ acceptance of any statistically significant improvement shown in a single randomized trial and lofty drug prices has created a situation where it is now, hypothetically, profitable for a company to run a clinical trials portfolio of chemically inert compounds,” he said in a 2018 comment he co-authored in Nature Reviews | Drug Development. “While the current cancer drug pipeline is certainly superior to inert drugs, we must rethink market incentives to encourage transformational drug development.”

He has also been critical of COVID vaccine mandates for adolescents and children. And he was critical of the agency’s accelerated approval of Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy Elevidys and the limited data to support the decision.

Though industry executives have been circumspect in their comments, long-time biotech journalist John Carroll, editor-in-chief of Endpoints News, seems to have reflected industry and investor sentiment in his response to the news in a post to the social media site X saying, “You’re f/$@ing kidding me?”

Investors responded in similar fashion, sending already beaten-down biopharma stocks lower and reducing the Nasdaq and S&P biotech indices by about 6 percent on the day of the news. Sarepta shares fell more than 25 percent on May 6. It then reported disappointing earnings and lowered its forecast for the year, which sent its shares down an additional 23 percent.

FDA Commissioner Makary recently excited the rare disease community when he said during an interview with Megyn Kelly on SiriusXM that the agency is considering a new pathway for rare disease drugs that could allow for an approval based on a “plausible mechanism.”

The rare disease community has long fought for the FDA to take steps to facilitate easier access to promising new therapies for people with rare diseases. FDA Commissioner Makary generated excitement when he recently said during an interview with Megyn Kelly on SiriusXM that the agency is considering a new pathway for rare disease drugs that could allow for an approval based on a “plausible mechanism.”

Makary may have offered encouraging words, but it’s actions that matter. Prasad’s predecessor, Marks, recognized the need for regulatory flexibility, particularly when it came to the development of therapies for smaller patient populations. Prasad’s appointment suggests that getting new therapies to people with rare diseases will be harder, and the introduction of any new mechanism for rare disease approvals seems less plausible.

Photo: Newly appointed Director of the FDA’s Center for Center for Biologics Evaluation and Research Vinay Prasad