The U.S. Food and Drug Administration (FDA) is a bureaucratic, risk averse organization. Its culture of risk aversion and skepticism was established back in 1960 when FDA drug reviewer, Frances Oldham Kelsey, famously refused to approve Thalidomide due to inconclusive data regarding safety testing. For her role protecting children from birth defects, she gained national prominence, receiving the highest federal employee civilian award from President Kennedy. Career bureaucrats at the FDA strive to continue her legacy.
The FDA’s first year under the second Trump Administration was off to a slow start. In 2025 the federal agency approved fewer drugs (39) than any other year for the past nine years except for one (37 in 2022). By contrast, in 2018 under Trump the agency approved 59 drugs.
The FDA under Trump has consistently maintained its stated goal of removing bureaucratic obstacles to drug approval. FDA commissioner, Marty Makary, announced steps to speed the approval of new drugs, including allowing one well-designed clinical trial rather than two or more trials to show efficacy. Yet, drug companies continue to claim inconsistent guidelines, endpoints changed and a skeptical agency when it comes to rare disease therapies. Recently drugmakers have complained the FDA moved the goalposts, after agreeing to them.
So, what is going on? Why, on the one hand does the FDA say it is willing to expedite approval of drugs, while also dragging its feet? For starters, senior FDA officials say they are willing to approve drugs quickly but refuse to rubber stamp drugs that failed to show promise. The following is from the New York Times:
[T]he agency has increasingly issued drug rejections or refusals — about 20 in the last eight months — that underscore a break from decades of general stability in the F.D.A.’s top ranks across several presidential administrations. It has advised companies to launch costly and complex studies that could add years to finding treatments for rare diseases with no cure.
On closer inspection there really is no dichotomy. The FDA’s chief science and medical officer, Vinay Prasad, rose to prominence in academia picking apart poorly constructed medical studies. He brings his analytical skills – his skepticism – to his job at the FDA. In a podcast prior to his FDA appointment he stated:
“I constantly see medicine that’s completely Hail Mary batshit crazy medicine,”
“I guess my concern is: How can we live in a world where society is taxed to pay for these unproven things?”
Dr. Prasad has also complained of “revolving-door politics,” suggesting that some FDA reviewers recommend approving drugs despite little evidence they work. Salaries at drug companies are double or triple that at the FDA. Many FDA scientists worry about offending drug companies because they hope for lucrative jobs with many of these same companies in a future career after leaving the FDA. Officials also worry about offending advocates for rare diseases by rejecting a drug that may help a small few in at least a small way.
The economics of drug discovery for rare diseases is the therapies only treat small populations. Thus, they tend to cost millions of dollars, sometimes offering false hope in the process. In other cases, they are merely a bad deal for taxpayers and individuals paying insurance premiums. It all goes back to Obamacare, which banned annual and lifetime limits on benefits. The sky is the limit nowadays. Although rare diseases are, well, rare. They are also common. More from the New York Times:
The F.D.A. estimates that about 30 million people, nearly one in 10 in the United States, has a rare disease. So the agency’s decisions have hit companies developing drugs for rare diseases in a particularly hard way, and rare-disease treatments make up a large share of drugs seeking approval.
Perhaps it is for the best that the FDA is willing to approve drugs quickly, but only those that involve well designed trials that show they work. The purpose of a clinical trial should not be to define endpoints that the clinical trial can claim to pass. Rather, it should be to establish endpoints that clearly measures the health benefits of a new drug. Removing bureaucratic obstacles to new drug approval should also expedite ineffective drug rejections, especially ones that cost millions of dollars per patient.
Read more at New York Times: F.D.A. Faces Upset Over Denials of New Drugs