FDA is expected to drop its long-standing requirement of two pivotal studies to receive approval for new drugs in an apparent effort to speed up availability of select medical products.

Moving forward, FDA will only be requiring one adequate and well-controlled clinical trial supported by confirmatory evidence to approve new drugs and other healthcare products, according to U.S. News.

The move, unveiled by FDA Commissioner Marty Makary and other senior officials, marks one of the most significant regulatory policy changes in decades, and also reflects continued efforts by the agency’s leadership to streamline reviews.

Why did FDA decided to change its new drug approval requirements?

Since arriving at the FDA last April, Makary introduced a series of initiatives aimed at reducing review times, including the National Priority Voucher Program and offering one-month reviews for drugs deemed to serve “national interests.”

In a policy article published Wednesday, Makary and FDA vaccine chief Vinay Prasad argued that advances in biology, trial methodology, and statistical science justify moving away from the long-standing expectation of two pivotal trials.²

“In this setting, over-reliance on two trials no longer makes sense,” they wrote. “In 2026 there are powerful alternative ways to feel assured that our products help people live longer or better than requiring manufacturers to test them yet again.”

Both officials expected the change would spur “a surge in drug development.”²

How long has FDA required two studies for new approvals?

According to U.S. News, the two-trial standard dates back to the early 1960s, when Congress required “adequate and well-controlled investigations” before new drugs could be approved.

For decades, the FDA interpreted that language as requiring at least two independent studies, a safeguard designed to reduce the risk that a single positive trial was a statistical fluke.¹

From a statistical standpoint, the requirement for two trials is expected to reduce the likelihood of a false-positive finding simply because a sponsor must demonstrate success more than once. However, regulators are now arguing that modern drug development rarely involves testing biologically implausible compounds, and instead, therapies are typically supported by detailed mechanistic science, biomarker data and increasingly sophisticated trial designs.²

Since 1997, the FDA has had explicit statutory authority to approve drugs on the basis of a single adequate and well-controlled study combined with confirmatory evidence. Such evidence can include mechanistic data, results in related indications, animal models, class effects, real-world evidence or, in some cases, a second trial.²

How does this change effect the review process?

Moving forward, rather than focusing on the number of trials, reviewers are expected to concentrate on trial quality, including magnitude of effect, appropriateness of control arms, endpoint selection, statistical power, blinding, handling of missing data, biological plausibility and alignment with intermediate biomarkers.¹

What are healthcare professionals saying about the change?

Dr. Janet Woodcock, who led the FDA’s drug center for roughly two decades before retiring in 2024, said the scientific reasoning behind the shift is sound, “The scientific point is well taken that as we move toward greater understanding of biology and disease we don’t need to do two trials all the time.”

Woodcock noted that oncology and rare diseases have already largely operated under single-trial flexibility, and that the greater impact may be on drugs for more common conditions that historically were expected to meet the two-study benchmark.¹

“It’s not the cancers and the rare diseases that will be affected by this,” she said. “The agency has been approving those on a single trial already.”

Makary and Prasad reject criticism that the new policy weakens standards. Both officials argue that two poorly designed trials do not guarantee valid conclusions, and that concentrating review resources on a single rigorous, well-designed study may actually strengthen regulatory scrutiny.²

“Without examination of the quality of a study, two trials may even provide a false assurance,” they wrote.

Sources

1. FDA Will Drop Two-Study Requirement for New Drug Approvals, Aiming to Speed Access U.S News February 18, 2026 https://www.usnews.com/news/business/articles/2026-02-18/fda-will-drop-two-study-requirement-for-new-drug-approvals-aiming-to-speed-access
2. One Pivotal Trial, the New Default Option for FDA Approval — Ending the Two-Trial Dogma The New England Journal of Medicine February 18, 2026 https://www.nejm.org/doi/full/10.1056/NEJMsb2517623