What should be the FDA’s mission: to crank out drug approvals or only approve ones that are safe and effective? The U.S. Food and Drug Administration (FDA) is on track to approve only 39 new drugs in 2025. That is the second lowest number since 2017. The lowest number was 37 in 2022 during the Biden Administration.
The number of drugs approved is a function of many things, some of which are beyond the control of the FDA. Primarily is the number of drug applications that are nearing the end of the process in any given year. Then there is the quality of the applications, quality of the data, and the complexity of the drug trials. Finally, there is the staffing, staff expertise, resources, and priorities at the FDA. The latter has seen some turmoil since the radical change in the status quo at the U.S. Department of Health and Human Services and (to a lesser extent) at the FDA itself. The FDA only has control over the priorities the agency sets.
Recently the Wall Street Journal highlighted some top officials expressing skepticism about some recent approvals of drugs of questionable effectiveness, saying:
Dr. Tidmarsh shares Dr. Prasad’s regulatory paternalism. He has criticized the FDA’s past leaders for “endangering patients” by approving drugs that “gave them false hope,” and “cost vast amounts of money that our health case [sic] system can ill afford.” In September he called for a reappraisal of the FDA’s accelerated approval process.
Indeed, two investigative journalists reviewed 400 drug approvals from 2013 to 2022 and found that nearly three-fourths (73%) failed to meet all four FDA scientific standards for safety and effectiveness. The following was reported in the Daily Telegraph:
Shockingly, 40 drugs met none of the criteria, which include control groups, replicated trials, participant blinding, and clinically meaningful endpoints.
The investigation highlights a pattern of regulatory failure, with many drugs fast-tracked through approval on preliminary or surrogate data, like tumour shrinkage, rather than actual benefits such as extended survival or symptom relief. Some follow-up trials were never completed; others revealed the drugs were ineffective or dangerous, yet the products remained on the market. High-profile examples include Copiktra, which was later linked to earlier death in cancer patients, and Elmiron, which caused vision loss in some users but is still prescribed today.
It is difficult to say whether recent scrutiny had anything to do with the slower pace of new drug approvals. Officials may not agree with the findings that nearly 300 drugs failed to meet all the basic science criteria or that 40 drugs met none. I have also been somewhat critical of expensive drugs that are largely ineffective beyond what would be expected of a placebo, saying:
A little over four years ago the FDA buckled under pressure and approved Aduhelm, a monoclonal antibody for early-stage Alzheimer’s disease. Supporters argued it was the only new Alzheimer’s drug in many years. However, critics later claimed it was a hyper expensive drug that barely worked with severe side effects (e.g., brain bleeds). The drug has since been withdrawn from the market.
A few months ago Charles Hooper and Solomon Steiner proposed that the FDA should assess safety rather than efficacy. A problem with that is that neither patients nor doctors can easily assess whether a given (new) is likely to be beneficial. Patients could spend months taking ineffective drug therapies, while consumers and their insurers could spend hundreds, possibly thousands of dollars on ineffective drugs. The economic incentive would reward quickly developed, ineffective snake oil elixirs and penalize the time it takes for actual drug discovery.
Sometimes the FDA is doing its job well when it declines to approve a new drug. Case in point is thalidomide. Sometimes it is doing its job when it takes the time to require quality data to support approval. Whether the FDA’s number of new drug approvals in 2025 is down due to doing its job well, slacking off or just short on staff is hard to know. In any case, the process should be streamlined to quickly weed out ineffective drugs and speed effective ones to market.