Any New Year’s resolution can be met if the bar is set low enough. The Food and Drug Administration (FDA) approved 55 novel drugs in 2023, up from 37 in 2022. While the FDA deserves kudos for allowing life-saving medications to come to market (e.g., Fruzaqla for colon cancer), the agency has continued to reject promising medications for dubious reasons. Patients with chronic cough, severe allergies, and various cancers are the latest victims of the FDA’s continued risk aversion. In 2024, America’s drug regulator should resolve to embrace flexibility, lower healthcare costs, and allow access to game-changing and money-saving treatments.
Over the past 70 years, the FDA has evaluated medications for “safety” and “efficacy” using the results of clinical trials performed by manufacturers. The problem is that, even in cases where medications show promise, the FDA is inclined to kick the can down the road (reject medications) and insist on more testing. The agency took this ridiculous approach to a medication called Neffy, which is a needle-free (nasal spray) alternative to EpiPens. EpiPens are used in life-threatening situations to treat severe allergic reactions. But, more than half of severe allergy sufferers are put off by EpiPen’s needle and bulky setup and opt not to carry the medication with them regularly.
Most patients surveyed said they wouldn’t have the same issue with neffy, which comes in slender packs and is easy to carry around. Most members of the FDA’s Pulmonary-Allergy Drugs Advisory Committee were swayed by sponsor ARS’ surrogate trial data and opted to endorse the product’s benefits in May 2023. But, the FDA turned around in September and insisted on more data before the product could come to market. For the millions of Americans with epinephrine prescriptions, the regulatory holdup will mean fewer options at a higher cost.
The FDA has embraced a similarly risk-averse approach to approving medication for chronic coughs. About 5 percent of adults in the U.S. have this deeply-disruptive condition, and its prevalence has significantly worsened since the start of the COVID-19 pandemic. A medication called gefapixant has already been approved in Europe and Japan to treat chronic cough, and clinical studies show statistically significant reductions in 24-hour cough frequency. Despite this promising evidence (and the fact that the FDA should have reciprocal approvals with Europe and Japan), the FDA has twice rejected the medication – most recently in December 2023. The FDA has not indicated it believes that gefapixant’s safety is a concern. Rather, the agency has broken ranks with its European and Japanese counterparts because of what it sees as mixed efficacy data. Regulators could have opted to approve the drug and closely monitor post-market data in the U.S., Japan, and Europe, or left it to insurers and patients to decide for themselves whether the medication is effective. Instead, it’s back to square one for patients. There remains no approved treatments for chronic cough in the U.S., and patients are left to wonder if they’ll ever get their quality of life back.
The FDA’s 2023 rejections are reminiscent of similar nit-picking in 2022. One poorly reasoned rejection centered around a medication called omburtamab, designed to treat a rare pediatric brain cancer. At first, the agency granted some leeway and allowed omburtamab’s sponsor (Y-mAbs Therapeutics) to give the drug to a small number of patients and compare survival outcomes to a historic dataset of similar patients who never had access to the drug. While impressive outcomes were reported, 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” dataset. Additionally, the dataset 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.” In the same analysis, though, the FDA reported that it was in fact able to control for patients’ use of other treatment (i.e., radiation therapy, surgery, chemotherapy) and the time-period of treatment. And, even after adding the controls, the results appear encouraging for the medication. The data suggests that patients taking omburtamab live 7-12 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.
The FDA needs to take a wider view of the evidence and embrace flexibility rather than risk-aversion. Medications such as neffy, gefapixant, and omburtamab could benefit millions of patients if the agency permitted their use. It’s time for a better, more responsible FDA in 2024.
David Williams is the president of the Taxpayers Protection Alliance.
