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The FDA recently gained more leverage over pharmaceutical companies. Will it use it?


After years of criticism, the Food and Drug Administration is getting new tools to hold drug companies accountable when it comes to speedy approval of their medicines. NPR's pharmaceuticals correspondent Sydney Lupkin reports. The agency has yet to show how it will put its new leverage to use.

SYDNEY LUPKIN, BYLINE: The huge spending bill that became law last December also included fixes to a problem that has dogged the FDA for years. Drugmakers could get fast approval for certain drugs with preliminary data on the promise that they would do more research after the fact to make sure the drugs worked. But companies were often slow to follow through on these so-called accelerated approvals, leaving patients and doctors uncertain about their medicines. Will the FDA now force drug companies to start their studies on time? I asked Robin Feldman, a professor at the University of California College of the Law, San Francisco.

ROBIN FELDMAN: This essentially strengthens their hand. However (laughter), just because the FDA has the power doesn't mean it's going to use it.

LUPKIN: There have been times when the FDA has had powers, but neglected to use them. Conversely, the agency has also attempted to put its foot down, gotten sued by drugmakers, and lost. She says the new law can come in handy if a drug company tries to push back on an FDA decision. The FDA wouldn't say whether it's doing anything differently on accelerated approvals other than that it's working on a plan to implement the new law. Here's Feldman again.

FELDMAN: Generally, these are discretionary rather than mandatory. In other words, it allows the FDA to do things but doesn't require the FDA to do things. You know, that's a big difference.

LUPKIN: We should note that the COVID vaccines were not granted this kind of approval, but accelerated approvals do apply to more than a hundred drugs approved to treat cancer, HIV, preterm birth and more over the last three decades. An NPR investigation last year found that many of the promised confirmatory studies are delayed, sometimes for years. The new law allows the FDA to require companies to start confirmatory studies before it grants accelerated approval, something the agency told me last year it didn't have the authority to do. Here's Dr. Reshma Ramachandran of Yale School of Medicine, who pushed for the fix.

RESHMA RAMACHANDRAN: We wanted that to be, like, codified in legislation. So that wouldn't be an exception; that would just be the rule. And I think FDA, you know, also made very clear when they put out a piece in New England Journal of Medicine, showing that when that happens, that prevents significant delays for starting the trials but also completing the trials.

LUPKIN: The law may also make it easier for the agency to take drugs off the market if studies find the drug is not effective. Here's Dr. Aaron Kesselheim, a professor of medicine at Brigham and Women's Hospital and Harvard Medical School.

AARON KESSELHEIM: When a drug is given accelerated approval and then that confirmatory study fails, that's really an important finding that indicates that that drug does not have the same level of effectiveness that we expect of other approved drugs. And there is no reason for that drug to remain on the market at that point.

LUPKIN: In addition, the law creates new transparency requirements and a counsel to get everyone at the FDA on the same page about how this kind of approval should be used. Still, Kesselheim says the changes may not be enough.

KESSELHEIM: The final language was not as airtight as it could be, you know, so I do think that there is wiggle room.

LUPKIN: So the rules may need to be tightened again in the future. Sydney Lupkin, NPR News. Transcript provided by NPR, Copyright NPR.

Sydney Lupkin is the pharmaceuticals correspondent for NPR.
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