FDA Abandons Two-Study Standard for New Drug Approvals to Accelerate Access
The Food and Drug Administration (FDA) is set to abandon its decades-old standard of mandating two rigorous clinical studies for the approval of new drugs. This significant policy shift, announced on Wednesday, 18 February 2026, aims to expedite the availability of novel medical products by reducing regulatory bureaucracy.
New Default Position: One Study Required
In a piece published in the New England Journal of Medicine, FDA Commissioner Dr. Marty Makary and top deputy Dr. Vinay Prasad outlined that the agency's "default position" will now require only one study for new drugs and other innovative health products. This change reflects the Trump administration's ongoing efforts to streamline drug approvals and enhance patient access to cutting-edge treatments.
"In this setting, overreliance on two trials no longer makes sense," wrote Makary and Prasad. "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."
Historical Context and Scientific Rationale
The two-study standard dates back to the early 1960s, when Congress passed legislation requiring the FDA to review data from "adequate and well-controlled investigations" before clearing new medications. For years, this was interpreted as necessitating at least two studies, typically involving large patient cohorts and extensive follow-up periods to confirm results were not anomalous.
However, since the 1990s, the FDA has increasingly accepted single studies for treatments targeting rare or fatal diseases, where large-scale trials are challenging. Over the past five years, approximately 60% of first-of-a-kind drugs approved annually have been cleared based on a single study, driven by congressional laws encouraging flexibility for serious conditions.
Dr. Janet Woodcock, the FDA's former drug director, supported the change, noting it aligns with the agency's gradual move toward relying on one trial supplemented by supporting evidence. "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," said Woodcock, who led the FDA's drug center for over two decades before retiring in 2024.
Impact on Drug Development and Industry
FDA officials predict this policy shift will spur a "surge in drug development," particularly for common diseases that previously did not qualify for reduced testing standards. Woodcock clarified that drugs for cancers and rare diseases, which have often been approved via single trials already, will see minimal impact. Instead, the change will primarily benefit treatments for more prevalent ailments.
Since assuming his role in April, Commissioner Makary has implemented several directives to shorten FDA reviews, including mandating the use of artificial intelligence by staff and offering expedited one-month reviews for drugs deemed to serve "national interests." These measures contrast with the agency's more cautious approach to other products, such as vaccines and gene therapies.
Contrasting Approaches and Industry Uncertainty
The new policy stands in stark contrast to recent FDA actions on vaccines and gene therapies. Last week, the agency's vaccine division, led by Dr. Prasad, initially rejected Moderna's application for a new mRNA flu shot due to insufficient clinical trial data, only to reverse course after Moderna agreed to an additional study in older individuals. Similarly, Prasad has rejected multiple experimental gene therapies and biotech drugs, citing the need for more definitive evidence.
This inconsistency has created confusion within the biotech industry, weighing on company stocks and conflicting with Makary's public emphasis on speed and flexibility. Woodcock cautioned that the true impact will depend on implementation. "Implementation will be everything," she stated. "Since the agency's approach is unclear, and the industry is already baffled, I don't think this adds any illumination."
The FDA's move to drop the two-study requirement marks a pivotal moment in pharmaceutical regulation, balancing the need for scientific rigor with the demand for faster access to innovative treatments. As the policy unfolds, its effects on drug development, patient care, and industry dynamics will be closely monitored.
