FDA Commissioner on Accelerated Approval, misinformation, and food

Accelerated Approval, growing misinformation and declining life expectancy, and the need for more resources on the food side of the agency were among the topics FDA Commissioner Dr. Robert M. Califf discussed at the 2022 BIO International Convention.

He joined BIO President and CEO Dr. Michelle McMurry-Heath for a fireside chat, with the crowd spilling into the overflow room in the final educational panel of the week.

In his second term as head of the Food and Drug Administration, he’s weathered a pandemic, attacks on the Accelerated Approval Pathway, and an infant formula shortage. He discussed these challenges, priorities for the next 12-18 months, and the legacy he hopes to leave behind.

Accelerated Approval is a ‘relay race’

“I’m a big fan of Accelerated Approval,” said the FDA Commissioner, but we need to think about it like a “relay race.”

“FDA shouldn’t stop trying to be the fastest team and the most efficient team on the first lap.” Still, we shouldn’t “just drop the baton at the end of the first lap and hope [Centers for Medicare and Medicaid Services] can find the baton and get going.”

We “need a smooth hand-off,” and an ability to “get things to market quickly but also find out really quickly what turns out to be effective.”

Nonetheless, he reassured the audience that FDA “is not responsible or accountable for cost,” and will continue to “look at safety and effectiveness, not cost.”

Accelerated Approval provides benefits to many patients—including Dr. Califf’s own mother, who he said got a few extra years thanks to an Accelerated Approval drug to treat her multiple myeloma.

However: “Making sure those confirmatory studies get done is really the critical thing,” he said, responding to Dr. McMurry-Heath’s question about Accelerated Approval in the FDA user fee negotiations. “The public is watching closely.”

Food, Animal & Veterinary centers need ‘a shot in the arm’

“The medical products side, in my view, is working pretty darn well,” he said, thanks in large part to the 21st Century Cures Act, which was designed to accelerate medical product development.

However, there hasn’t been a similar effort on the food side, which he said is “chronically underfunded” and needs a “shot in the arm like 21st Century Cures.” The infant formula shortage shows the importance of this: “It’s easy to deal with the crisis of the day and not think about preparing for the future,” he noted.

“The center for veterinary medicine is the most under-appreciated part of FDA,” he added, which impacts food, animal health, and pet food. With advances in “technology-driven foods” and nutrition in the era of big data, he’s “excited about what can be done if we get this right.”

Misinformation is ‘the leading cause of death’

“Almost all the COVID deaths now are preventable” if patients were up-to-date on COVID-19 vaccines and boosters and had access to antivirals, said the FDA Commissioner.

Misinformation is “the leading cause of death in the United States today.”

This is related to the decline in life expectancy in the United States, which is “not because of some mysterious problem but because of chronic common disease,” including heart disease, diabetes, and mental health. “For most of these things, we do have effective treatments already—they just need to be used better.”

“There’s little dispute that America is innovating for the world,” so it is paradoxical that U.S. life expectancy is so low, especially compared to other wealthy nations.

Another priority: evidence generation

It’s important to close “the gap between the point at which the FDA approves a treatment for marketing, and then what’s known about how to use that treatment, how it should be valued,” he said.

We should fill that gap with “an evidence-generation system that gives the information that’s really needed to make good decisions going forward.”

During the conversation, he noted the importance of real-world data and the role of technology in helping to collect it, international data (“of course” FDA is interested in collecting it), and clinical trial diversity.

When asked what legacy he hopes to leave at FDA, after serving his second term, Dr. Califf responded “leaving the FDA in a better position than it was in to optimize public health.”

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