The FDA will make a single pivotal clinical trial the default for approving new drugs, agency leaders said in the New England Journal of Medicine. Officials argue that advances in trial design and biological understanding reduce the need for a universal two-trial requirement and could speed development and patient access. Former FDA drug chief Dr. Janet Woodcock welcomed the move but warned that implementation and consistency across the agency will determine its impact.
FDA Adopts One Pivotal Trial As The Default For New Drug Approvals To Speed Access
Dr. Marty Makary, commissioner of the Food and Drug Administration speaks at an event on addiction recovery in the Oval Office of the White House, Thursday, Jan. 29, 2026, in Washington, as Attorney General Pam Bondi and Interior Secretary Doug Burgum listen. (AP Photo/Allison Robbert)
The Food and Drug Administration announced a major policy shift: the agency will make a single pivotal clinical trial the default standard for approving new drugs and innovative health products. FDA Commissioner Dr. Marty Makary and senior official Dr. Vinay Prasad outlined the change in an essay in the New England Journal of Medicine, saying advances in research methods and biological understanding allow greater confidence without always requiring two trials.
What Changed—and Why
For decades, the FDA has typically expected at least two well-controlled studies to demonstrate a new drug's safety and effectiveness. That standard dates back to a 1960s law directing the agency to rely on "adequate and well-controlled investigations." The second trial historically served to confirm that initial results were reproducible rather than chance findings.
Makary and Prasad argue that modern trial design, better biomarkers, improved statistical methods and deeper biological insight mean the two-trial expectation is often unnecessary. They say the agency's "default position" will now be to require one high-quality pivotal study, supplemented by supporting evidence when needed, to accelerate patient access to promising treatments.
Context And Trends
Regulators have already relaxed the two-trial expectation in certain circumstances. Since the 1990s the FDA frequently accepted single-study approvals for rare or life-threatening conditions where enrolling large numbers of patients is difficult. In recent years, roughly 60% of first-of-a-kind drugs approved each year were cleared based on a single trial, reflecting statutes and policies that allow flexibility for serious or hard-to-treat diseases.
Dr. Janet Woodcock, the FDA's former director of the drug center, said the update reflects an existing trajectory at the agency. '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,' she said, while cautioning that how the policy is implemented will determine its real-world impact.
Internal Tensions And Implementation
The announcement comes amid apparent differences in approach inside the agency. The FDA's vaccine division recently declined to accept Moderna's application for an updated mRNA flu shot, calling its clinical trial insufficient; after Moderna agreed to run an additional study in older adults, the agency said it would review the submission. Separately, agency reviewers have asked for more evidence on several experimental gene therapies and biotech drugs—decisions that have at times pressured biotech stocks and raised questions about consistency.
Makary and Prasad predict the new default will spur a surge in drug development and faster patient access, but experts emphasize that specifics of implementation, the standards for supporting evidence, and how review divisions apply the policy case-by-case will be decisive.
Reporting for this article included material from The Associated Press. The AP is solely responsible for all content.
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