Califf: AI, Drug Shortages, Cell and Gene Therapies Are Among FDA Priorities

Artificial intelligence (AI), drug shortages and cell and gene therapies are just a few of the areas on which the FDA is focusing during a major reorganization. Those were some of the topics that FDA Commissioner Robert Califf, M.D., discussed with Mike Tuffin, president and CEO of AHIP, at the insurer trade group’s 2024 Medicare, Medicaid, Duals & Commercial Markets Forum, held March 12 through 14 in Baltimore.

When asked his vision for the FDA in 2024 and beyond, Califf responded that 2024 priorities were different because it’s an election year, and the agency’s priorities are “tied to the administration that’s in place.”

For 2024, the “most important” issue is food. During the March 13 session, titled “FDA Policy Priorities for 2024,” Califf noted that the agency was in the midst of its largest reorganization ever, and that will include a “heavy focus on nutrition but also the safety of the food supply,” which is impacted by an “amazingly complicated” supply chain.

He also highlighted two big topics on the medical side: gene editing and AI, the latter of which “is affecting everything we do.”

During his prior stint as FDA commissioner under former President Barack Obama from February 2016 to January 2017, the agency was putting in place a framework for AI and machine learning (ML). Compared with that time, he said, “I feel a lot like a semi-expert in the area” of AI. His current job “is mostly to say [to agency engineers], ‘Here’s what you’ve got to do. It’s probably not going to work, because many things don’t work that we try to implement.’”

That said, developments in the field have been occurring, including digitization and “profound computational capacity,” which companies such as Apple, Microsoft, Amazon and Google are currently building. “Now we’re in an exponential phase of incorporation of AI into our lives.” The easiest way to describe it, Califf said, is the evolution from people driving “around with maps in their glove box” to navigate to printing out MapQuest directions. And “now you just get in your car and talk to your car.”

With medical AI, he said, many people contend that there has to be an understanding of exactly how it’s working. However, “I don’t think many people here could actually describe what happens between a road and your car to get that decision tree into your car. But because it’s so profound, there can be tremendous good, or there can be tremendous harm depending on how AI is used, and there are a lot of conflicting views about how it should be regulated.”

“No one wants, for example, catastrophically bad decision support to end up in a medical place,” he asserted. He compared doctors with drivers in that “they’re not going to stop and question how does AI work? But if it begins to work for you, it’s going to be incorporated into the workflow, which I think will be great.”

The FDA has a scheme dating back to 2014 and 2015 that says, “if you’re going to implement AI, you have to monitor it; you have to have something like postmarket surveillance of drugs and devices, where you’re constantly watching what that algorithm is doing,” he explained. Monitoring is particularly important with generative AI, which sometimes “invents things.”

According to Califf, “you might think” that within the health care system, entities are in sync on two issues: patient outcomes and finances. “Optimizing finances is so much easier than optimizing outcomes. A big worry is that AI is going to be used to optimize finances that will actually divert from what’s best for patient outcomes,” and there have been some instances in which this has occurred. “It’s not a new dilemma; it’s existed for a long time.…The clinical community feels under siege to just generate more revenue at the expense of really looking after patients.”

“We are fully supportive of mechanisms that move us toward making sure the incentives are aligned across the system and are aligned” on patients, remarked Tuffin.

“It’s so frustrating because I think we all agree on that, but somehow…we’re all well paid, and yet we are somehow victims of a system where…the vector of financial success is actually divergent from the vector of what’s best for patients,” responded Califf. “I think personally, we’ve got to figure this out.”

FDA Will Address Trust Issues, Drug Shortages

Asked about how the agency thinks about “the collapse of trust in institutions in the country [and] the spread of misinformation and disinformation when it comes to health and safety,” Califf replied that “it starts at the core of what the agency does. You know the old saying ‘if you want to be trusted, be trustworthy,’ and it’s hard for people to trust if you’re expressing everything in terms of language that people don’t understand.”

To address this, the FDA has a “big focus” on “speaking in plain language and educating and training our scientists to be more publicly outgoing.”

Noting that pharmacists, doctors and nurses always seem to rank higher in terms of trustworthiness than do scientists, federal agencies and politicians, Califf underscored the need to “reestablish much tighter relationships…with primary care.”

Tuffin then asked about the FDA’s role in the ongoing issue of drug shortages, and Califf explained that there are three types currently:

(1) Generic drugs: The reason for this is that “we’re paying too low for generic drugs,” he explained. The current situation means that “the lower the price of a generic, the higher the likelihood or probability of a shortage,” he said, and much of this is tied to the global supply chain, which can be a bit of a black box. It’s “going to take years” to fix this market, he asserted.

(2) Drugs for attention-deficit/hyperactivity disorder (ADHD): “The amount of manufacturing is set by the DEA [Drug Enforcement Agency] because those drugs are addictive.…The DEA quota in the U.S. affects the global distribution of these” agents, and demand for them is rising, he observed. While the FDA has a role to play, it is a bit different than in other areas.

(3) Weight-loss drugs: “What I thought I would never see,” he said of that shortage. “Who would have thought that there would be a drug that could make an enormous amount of money,…but we can’t make enough to fill that desire?”

“These are three very different categories, and the biggest one” expected over the next few years is low-cost generics, he maintained. “Ninety-five percent of prescriptions are generics now in the U.S. We’re paying too much for innovator drugs and too low for the low-cost generics.”

Cell and Gene Therapies Pose ‘Big Societal Issue’

The implications of emerging cell and gene therapies are “a big deal,” Califf asserted. “It couldn’t be more exciting.…We now have 10,000 rare diseases with no effective treatment,” and many of them kill children at a young age.

“Most of these treatments are not going to be curative, but there will be children who will live a long time who would have died” without them.

For many of these therapies, “it’s just going to be a matter of how much money we can put in the R&D, what the R&D capacity is and our ability to organize the clinical trials. But they’ve got to figure out what’s a model for a sustainable industry?…It’s a big societal issue that we need to solve.”

Another challenge is that the drug development process is difficult. “Ninety percent of drugs in Phase I trials don’t make it to market…because they either don’t work or have toxicity,…so we also need your help with clinical trials,” Califf told the audience.

Finally, Califf said that he did not expect any “major changes” to the accelerated approval process. “I think we have all the tools we need to accelerate approval,” including some new tools just this year with respect to enforcing confirmatory studies.

Imploring the audience’s help, Califf noted that “one thing we hear from clinicians about participating in clinical trials is that they’re under so much pressure to generate revenue by seeing more patients over a shorter period of time” that this, combined with disagreements over “routine clinical care costs” with insurers, often disincentives them from participating in these trials.

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