FDA will drop two-study requirement for new drug approvals, aiming to speed access

WASHINGTON– The Food and Drug Administration plans to abandon its longstanding standard of requiring two rigorous studies to gain approval for new drugs, the latest change from Trump administration officials pledging to speed up the availability of some medical products.

Going forward, the FDA’s “default position” will be to require study of new drugs and other novel health products, FDA Commissioner Dr. Marty Makary and a top deputy, Dr. Vinay Prasad, wrote in a New England Journal of Medicine article published Wednesday.

The announcement is the latest example of Makary and his team changing long-standing FDA standards and procedures with the stated goal of cutting bureaucracy and speeding the availability of new drugs.

Since arriving at the agency last April, Makary has issued a series of directives that he says will reduce FDA reviews, including mandating the use of artificial intelligence by staff and proposing month-long reviews for new drugs that serve “national interests.”

This contrasts with the FDA’s more restrictive approach to other products, including vaccines.

In their paper published Wednesday, Makary and Prasad say dropping the two-trial requirement reflects modern advances that have made drug research “increasingly precise and scientific.”

“In this context, an overreliance on two trials no longer makes sense,” they write. “In 2026, there are other powerful ways to ensure our products help people live longer or better than requiring manufacturers to retest them. »

FDA officials predicted the change would lead to “an acceleration of drug development.”

Dr. Janet Woodcock, former FDA drug director, said the change made sense and reflected the FDA’s decades-long move toward a single trial, combined with supporting evidence, for a variety of life-threatening diseases, including cancer.

“The scientific point is well understood: As we move toward a better understanding of biology and disease, we don’t need to do two trials all the time,” said Woodcock, who led the FDA’s pharmaceutical center for more than 20 years before retiring in 2024.

The two-study standard for drugs dates to the early 1960s, when Congress passed a law requiring the FDA to review data from “adequate and well-controlled investigations” before authorizing new drugs. For decades, the agency interpreted this requirement to mean at least two studies, preferably with a large number of patients and significant follow-up duration.

The reason the second study was required was to confirm that the results of the first trial were not a fluke and could be reproduced.

But starting in the 1990s, the FDA began accepting more and more single studies for approval of treatments for rare or deadly diseases that companies often struggle to test in large numbers of patients.

Over the past five years, approximately 60% of new drugs approved each year have been authorized on the basis of a single study. The change reflects laws passed by Congress that directed regulators to be more flexible when reviewing drugs for serious or difficult-to-treat conditions.

Woodcock said the new policy announced Wednesday will primarily impact drugs intended to treat common illnesses that were previously ineligible for reduced testing standards.

“It is not cancers and rare diseases that will be affected by this,” she stressed. “The agency has already approved these on a single trial.”

The latest approach from FDA leaders contrasts with the agency’s recent actions on vaccines, gene therapies and other treatments.

Last week, the FDA’s vaccine division, led by Prasad, refused to accept Moderna’s application for a new mRNA flu vaccine, saying its clinical trial was insufficient. Then on Wednesday, the agency reversed course, saying it would review the vaccine after Moderna agreed to conduct an additional study on older adults.

Separately, Prasad has rejected a series of experimental gene therapies and biotechnology drugs, citing the need for additional studies or more definitive evidence. This trend weighed on the shares of many biotechnology companies and clashed with Makary’s public statements promoting the speed and flexibility of FDA reviews.

Woodcock said the pharmaceutical industry will have to wait and see if the FDA’s approach to promising experimental therapies changes.

“Implementation will be key,” she said. “Given that the agency’s approach is unclear and the industry is already confused, I don’t think this adds any insight.”

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