FDA drug approvals rest on fewer trials, and the cracks are showing
Novel FDA approvals since 2016 lean on fewer studies and more industry funding — and an MHRA suspension, tightening research borders, and a long Covid result show what that costs.
The bargain at the heart of drug approval has always been plain: prove it works, then prove it again. Regulatory frameworks in the US and Europe typically require at least two well-controlled studies before a product's effectiveness counts as validated — a safeguard against results that look convincing but are really chance. It is an unglamorous rule. It is also the rule this week's health reporting suggests is quietly fraying.
A retrospective review of every novel drug the FDA approved from 2016 through 2024, built from FDA public records and the ClinicalTrials.gov database to track how many completed trials stand behind each approval and who paid for them, found that novel approvals in the US have been supported by a decreasing number of studies since 2016. The trend, as Neurology Advisor summarized it, points to a system that brings new medications to market by relying on fewer clinical trials and more industry funding.
Hold those two threads together: fewer studies per drug, and more of the money behind those studies coming from the companies selling the product. Neither fact is a scandal on its own. Together they describe an evidence base growing thinner per approval at the very moment its funding grows less independent. The rest of the week's health news reads almost like a stress test of what that means.
When the floor is one study
Start with the starkest case. The Medicines and Healthcare products Regulatory Agency has suspended the new use of the drug avacopan after concluding that the pivotal trial data behind it is no longer reliable. One pivotal clinical study underpinned the license authorization, and that study was found to have compromised integrity and reliability.
The detail worth sitting with is what gets lost. Patients and providers lose access to a treatment that, as Medscape's report put it, showed real-world efficacy but can no longer be supported by regulatory benefit-risk standards. The drug did not stop working. The evidence did.
If a single pivotal study is the floor beneath a medicine, that floor is only as good as the study beneath it. The FDA review says the floors are getting thinner. The avacopan suspension shows what a collapse costs — and who pays: the patients who had a working treatment, and now do not.
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