The Food and Drug Administration’s Dr. Robert Califf called on insurers Thursday to help ensure that studies on new drugs in the accelerated approval pathway are still completed.
“I don’t think it’s just a matter of the FDA making sure the studies get done. This is a community issue where you all have a role to play,” Califf, commissioner of food and drugs at the FDA, said during a session at the AHIP Medicare, Medicaid, Duals & Commercial Markets Forum.
The accelerated approval program, created in 1992, is intended for drugs that aim to treat serious conditions that currently lack effective treatment and helps these drugs get approved faster. They are able to receive accelerated approval by demonstrating a “surrogate endpoint,” or an endpoint that could indicate real clinical effects but is not guaranteed.
After receiving accelerated approval, drug sponsors are supposed to conduct post-approval studies to confirm that the drug is in fact clinically effective — though it appears that this is not happening in every case. More than one-third of accelerated approval drugs had incomplete confirmatory trials, a 2022 report from the Office of Inspector General found.
Via the Consolidated Appropriations Act signed into law in December, the FDA now has more control over the drugs in the accelerated approval pathway.
“There are many details but the fundamental issue is the FDA has more authority now to require that the follow-up study gets started before the decision about approval,” Califf said. “Secondly, if the follow-up study is negative, streamlining the ability to take the product off the market, because that’s been another problem.” The process of removing drugs from the market is often difficult and therefore isn’t done frequently, he added.
Despite the new power given to the FDA, there are still some barriers with getting these studies completed, including from insurers, Califf said.
“I’m not aware of a major effort by the insurance plans to help people get studies done,” he stated. “In fact, what I’m hearing from clinicians out there is just the opposite, that it’s very hard to do research in the current environment, there are a lot of negative incentives. … What I’m hearing from doctors is they have no time to participate and they’re under intense pressure and many, many barriers from the insurance industry to get the studies done. … We certainly used to be able to blame technology. ‘It’s just hard to do it. You have to collect all this data, everything’s fragmented.’ Now we have electronic health records. We have claims, it’s all there.”
Completing the studies can also support the shift to value-based care, Califf said.
“It’s hard to assign value unless you’ve done the right studies … [Insurers], make it easier for health systems and clinicians to do the right study so we do get the outcomes,” he urged.
Ultimately, having complete research will rule out the drugs that don’t work and bring down costs, he declared.
“I really urge [insurers] to get beyond being a passive recipient of research and figure out what policies you can institute that actually make it easier to generate the evidence that we all need,” Califf said. “I believe, fundamentally as an amateur health economist, that if you spend more of your time generating evidence, you could stop paying for a lot of things because it would be clear that they don’t work. And ultimately, your costs will go down.”
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