2025 Data Highlights FDA Failures

This op-ed was originally published in Baltimore Sun.

Listening to Food and Drug Administration (FDA) leadership, it’s easy to think that 2025 was a banner year for patient choice. On December 19, Food and Drugs Commissioner Dr. Marty Makary listed the FDA’s accomplishments in 2025, including reducing decision times, hitting drug target date goals, “accelerating cures,” “cutting red tape,” and ushering in “a new era of innovation.” Tellingly, the agency’s leader failed to confront concerns that the FDA is becoming more risk averse and eager to reject new—and even existing—medications.

The unfortunate truth is that the agency has lost its way and shuns innovation. As a result, frustrated Americans are increasingly rebelling against the sclerotic agency by turning to gray and black markets for unproven “Chinese peptides” and other drugs. Commissioner Makary can only succeed in “accelerating cures” by boosting agency approvals and greenlighting lifesaving new medications. This can be accomplished without compromising safety.

According to a recent analysis by Nature, FDA approvals appear to be plateauing. The agency’s Center for Drug Evaluation and Research (CDER) approved 46 new therapeutic agents in 2025, in line with its five-year average. While year-over-year approvals are steady, the numbers of drugs entering clinical trials is anything but steady. As the biotech data platform RxDataLab notes, the number of active “Investigational New Drugs”—which have not yet been approved but are undergoing trials—surged from around 11,000 per year in the five years before COVID to more than 14,000 in recent years. The pipeline of drugs awaiting approval has become drastically more crowded, yet the FDA is approving about the same number of drugs it approved ten or eight or six years ago. Meanwhile, consultancies that track FDA-related trends report that the percentage of drugs receiving Complete Response Letters (i.e., rejections) in 2025 surged significantly higher than the 10-year average.

A few recent examples of the FDA’s approach to drugs show how bad the agency’s risk aversion has become. The FDA has repeatedly refused to greenlight an ophthalmic version of the already-approved Avastin for wet age-related macular degeneration. The agency (wrongly) claims that there isn’t enough evidence that the medication works, a contention that even notoriously risk-averse European regulators reject. But what’s good enough for German and United Kingdom patients apparently isn’t good enough for their transatlantic cousins.

Ditto for a higher dose formulation of Spinraza for the treatment of spinal muscular atrophy. According to a September report in Pharmaceutical Technology, “The agency chose to decline the antisense oligonucleotide’s (ASO) approval due to insufficient technical information listed under the chemistry, manufacturing and controls (CMC) section of its supplemental new drug application.” In other words, paperwork took precedence over a medication proven to be safe and effective. Never mind that the purportedly missing information is “readily available,” and Japanese consumers have ready access to the high dose Spinraza regimen.

U.S. regulators similarly snubbed Ebvallo, used to fight a rare blood cancer that is fatal if left untreated. The FDA has been reluctant to even allow clinical trials for the medication, and not because of any safety or efficacy concerns. Rather, the agency is hung up on fixable deficiencies at third-party manufacturing sites. Somehow, this hasn’t been an issue for European patients, who have had access to the lifesaving medication since 2022.

The FDA ended 2025 with yet another unwarranted rejection, this time for relacorilant. The drug is designed to treat Cushing’s syndrome, a rare hormonal disorder that can be fatal without medical intervention. Even though the relevant trial evidence showed significant improvements in patients’ blood pressure, fasting glucose, and weight with minimal safety issues, the agency is insisting on additional efficacy data. The result is patients and their doctors being denied the choice to weigh the evidence for themselves and make an informed choice.

The FDA can save and improve countless lives by truly “accelerating cures” and “cutting red tape.” The current approach just isn’t working for millions of Americans suffering from treatable diseases.