The Food and Drug Administration is on a roll. America’s drug regulator recently approved Veopoz as the first treatment for Chaple disease, a very rare inherited immune condition. The FDA also gave full approval to a drug called Leqembi, which slowed Alzheimer’s patients’ functional and cognitive decline by 27% over 18 months, according to trial data. And agency officials green-lit the first vaccine to protect infants from respiratory syncytial virus.

With all of these approvals, the FDA is on track to approve more novel drugs by the end of September than it did for all of 2022 . But challenges remain, and the FDA stubbornly clings onto a regulatory model grounded in risk aversion. It’s time for the agency to embrace its winning streak and keep innovation in the driver’s seat.

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Like many government agencies, the FDA’s approach to industry reflects caution and the prevailing panics of the day. During the e-cigarette (or vaping) product use-associated lung injury scare of 2019-2020, the agency (and Trump administration) was quick to blame vaping for dozens of deaths and thousands of hospitalizations, rather than black market products . The fiasco resulted in prohibition, overregulation, and general fear-mongering.

Unfortunately, the same logic too often applies to drug approvals. The agency is especially reluctant to approve drugs for rare diseases because of the inherent difficulty of finding a large number of volunteers for clinical trials. By the FDA’s logic, this makes the risk of generalizing findings unacceptably high even if the benefit would be to treat a devastating, terminal illness.

This mindset contributed to the 2022 rejection of a drug called omburtamab , designed to treat a rare pediatric brain cancer.

To the FDA’s credit, the agency allowed omburtamab’s sponsor (Y-mAbs Therapeutics) to give the drug to a small number of patients and compare survival outcomes to a historic data set of similar patients who never had access to the drug. But while impressive outcomes were reported by the company, regulators were skeptical because the patients being given omburtamab also had access to other treatments that were out of reach for at least some of the patients in the “control group” data set. Additionally, the data set contained some information collected during the 1990s and early 2000s, when cancer treatments may have been less effective.

The agency extensively communicated these concerns with its advisory committee, concluding that the FDA “cannot reliably attribute the observed [overall survival] OS difference to omburtamab.” Yet in the same analysis, the FDA reported that it was in fact able to control for patients’ use of other treatments (i.e., radiation therapy, surgery, chemotherapy) and the time period of treatment.

Even after adding the controls, the results appear encouraging for the medication. The data suggest that patients taking omburtamab live 7 to 12 months longer than their non-medicated peers. Despite these sustained positive findings, the advisory committee bought into the FDA’s critical briefing and voted to reject the drug . The FDA followed suit and sent Y-mAbs a rejection letter.

Unfortunately, the FDA is unlikely to change its attitude toward unconventional study designs anytime soon. Despite repeatedly urging more treatments for rare diseases, the agency has been critical of clinical trials using historical or “retrospective” data for controls. This retrospective data will play a larger role in drug evaluations going forward as diagnoses and treatments become narrower and more precise.

Even if advisory committees become more accepting of historic data collection, the FDA has indicated it’s not afraid to reject drugs lauded by experts. According to a 2019 analysis published in the Milbank Quarterly, “[t]wenty-two percent of the FDA's final actions were discordant with the advisory committee's recommendations. Of these, 75% resulted in the FDA making more restrictive decisions after favorable committee recommendations.”

The FDA will have to overcome its risk aversion and fear of less-than-perfect data sets to keep up its approval streak. Too many lives hang in the balance for the agency to lapse back into its old and restrictive mindset.

David Williams is the president of the Taxpayers Protection Alliance.