Lisa Jarvis: FDA appointee is a drug critic with a lot to prove

The controversial appointment of oncologist Vinay Prasad — an outspoken critic of the pharmaceutical industry and U.S. health agencies — to a key role within the Food and Drug Administration was a shock for drug companies. Biotech stocks immediately fell over fears that the bar for drug approvals suddenly got a lot higher.

Tightening regulatory standards is a reasonable goal. But achieving it requires delicately balancing evidence and compassion, weighing certainty against speed. Prasad has had a lot to say over the years about how companies and the FDA have gotten that balance wrong. Now, as the director of the Center for Biologics Evaluation and Research, which oversees vaccines, gene therapies and the blood supply, he has to prove that he can get it right.

Some of his more rigid and contrarian stances, such as his views on COVID vaccines, do not inspire confidence. Prior to his appointment, Prasad served as a professor at the University of California San Francisco. He gained notoriety during the pandemic with his near-constant haranguing of the Biden administration’s decisions. He was particularly incensed by recommendations to mask and vaccinate children, views that neatly align with those of FDA Commissioner Marty Makary and National Institutes of Health Director Jay Bhattacharya.

But before that, he was known for his criticism of the lack of rigor in oncology drug data. He’s been a social media gadfly, calling out what he sees as subpar studies and a low bar for approvals. He’s argued against faster approvals for cancer drugs based on measures like a tumor shrinking or the disease not worsening, which Prasad and others have shown don’t always translate into people living longer. When taking down a medical finding, one of his favorite responses is, “Randomize or STFU.”

That message, though acerbic, isn’t wholly controversial. He thinks the public deserves drugs that are safe and effective and the decisions around approvals should be free from conflicts. And many experts, including former regulators, bioethicists and health policy experts agree with his belief that too many drugs are making it onto the market before being proven to have real value.

I’ve also criticized the FDA for leaning too far into speed over certainty: Approving drugs on scant evidence doesn’t just give patients false hope, it can cause real harm. Patients risk significant financial hardship for a product whose benefits are modest at best or nonexistent at worst and might even have dangerous side effects.

And yet, those of us on the outside also have the luxury of armchair regulating. The FDA is often weighing data that might look promising but isn’t definitive, against patients’ urgent need for better treatments. “None of these decisions are going to be easy ones, where there is a single, objectively right answer,” says Holly Fernandez Lynch, a professor of medical ethics and health policy at the University of Pennsylvania's Perelman School of Medicine. Making the right call means engaging with every stakeholder in order “to be flexible where flexibility is appropriate, and push back where flexibility is inappropriate,” she says.

Not all drugs will fall under Prasad’s purview (many of the cancer drugs he has criticized, for example, were reviewed by the FDA’s Center for Drug Evaluation and Research). But he undoubtedly will be faced with deciding the fate of treatments for people with no other hope. Anyone who has listened in on advisory meetings for treatments for rare diseases like ALS or Duchenne muscular dystrophy will tell you that parent and patient testimonials can be gut-wrenching.

The gravity of those decisions calls for a degree of humility on the part of an FDA leader. And that’s the rub with Prasad: his public persona — often harsh and operating in the extremes rather than acknowledging the nuances — does not suggest he will strike the right tone in either his actions or words.

Ultimately, Prasad’s approach to regulating carries significant consequences for biotech and pharma companies, many of which are working on some of the most promising technological advances of the last two decades, including gene therapy and mRNA vaccines. Already many of those companies are struggling to come up with a viable business model for their products, which address very small patient populations. Raising the regulatory bar too high could push some out of the game entirely.

“If you make innovation too daunting, there’s going to be very little capital to fund it — and to find the next big breakthrough,” says Cantor Fitzgerald biotech analyst Josh Schimmer.

That doesn’t mean discoveries will screech to a halt, rather they will happen elsewhere, like China, he adds.

In his first meeting with FDA staff, Prasad reportedly struck a more even-keeled tone on the topic of rare disease drugs. He noted that “randomized controlled trials are not always necessary,” according to Inside Medicine, a Substack run by physician and public health researcher Jeremy Faust. And he apparently acknowledged that, “Evidence must also contextualize the condition — how rare and dire it is, and we should be flexible for the many people who do want to try things.”

While any glimmer of measured leadership is welcome, we should take it for what it is: a few rational sentences amid volumes of antagonistic posts on social media. We won’t truly know his threshold for approval until decisions start rolling out. That means for the foreseeable future, every advisory committee meeting and approval deadline for this critical corner of the FDA will be “big, binary events for the whole industry, each one a glimpse into where the bar is set now,” Schimmer says.

Even if he strikes a good balance, there’s another critical unknown to consider: Can he lead? Unlike his predecessors, Prasad does not have experience navigating the FDA. And he is walking into a downtrodden agency. Sweeping cuts at the Department of Health and Human Services did not spare the FDA, and even as some employees were called back, weeks of anxiety have taken a toll on morale. Many of those left behind, including experts who review new products, are looking for new jobs.

And like with the entire slate of COVID contrarians leading our health agencies, there’s the lingering question of who will really call the shots on hot button issues like vaccines. If HHS Secretary Robert F. Kennedy Jr. tries to interfere with products that fall under Prasad’s regulatory umbrella, will he push back? Some of his recent comments about the childhood vaccine schedule and the committee responsible for it veer too close to Kennedy’s views for comfort.

That could immediately affect fall COVID boosters, which Prasad now oversees. A post on his Substack suggests he is on board with recent comments by Makary suggesting updated vaccines need new studies, a position that would make it near impossible to get new shots authorized. “Folks who think COVID-19 vaccines should continue to roll out without randomized trials are anti-vaccine, anti-science, and pro-corporate,” Prasad wrote.

The concern now centers on a current threat, but we should also worry about the standard Prasad would set for future vaccines. His predecessor is credited with developing a clinical strategy that enabled COVID vaccines to be rolled out with breathtaking speed, ultimately saving millions of lives. How would Prasad treat the bar for authorization in an emergency?

All of this adds up to an intense period of uncertainty. The FDA has never been perfect or made everyone happy. But everyone seemed to largely understand the rules. With Prasad, that standard seems much less clear — and patients and industry alike will feel the impact.

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This column reflects the personal views of the author and does not necessarily reflect the opinion of the editorial board or Bloomberg LP and its owners.

Lisa Jarvis is a Bloomberg Opinion columnist covering biotech, health care and the pharmaceutical industry. Previously, she was executive editor of Chemical & Engineering News.