WASHINGTON — Federal health regulators are abandoning a decades-old practice that required pharmaceutical companies to conduct two comprehensive clinical trials before winning approval for new medications, marking another significant policy shift under the Trump administration’s push to accelerate medical product availability.
The Food and Drug Administration announced that moving forward, agency officials will typically demand just one clinical study for new medications and innovative health treatments, according to FDA Commissioner Dr. Marty Makary and deputy Dr. Vinay Prasad, who detailed the policy change in Wednesday’s New England Journal of Medicine.
This represents the most recent example of Makary and his leadership team overhauling established FDA protocols and standards, with officials citing goals to eliminate regulatory red tape and fast-track new treatment options for patients.
Following his appointment to the agency in April, Makary has implemented multiple policy changes designed to reduce FDA review timelines, including requiring staff members to utilize artificial intelligence tools and establishing expedited one-month review processes for drugs deemed important to “national interests.”
The new approach stands in stark contrast to the FDA’s stricter policies regarding other medical products, particularly vaccines.
In their Wednesday publication, Makary and Prasad argued that eliminating the dual-trial mandate reflects contemporary scientific progress that has made pharmaceutical research “increasingly precise and scientific.”
“In this setting, overreliance on two trials no longer makes sense,” the officials 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.”
FDA leadership anticipates the policy modification will trigger “a surge in drug development.”
Dr. Janet Woodcock, who previously directed the FDA’s drug division, endorsed the change as logical and consistent with the agency’s gradual shift over recent decades toward accepting single trials supported by additional evidence, particularly for life-threatening conditions like cancer.
“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,” stated Woodcock, who oversaw the FDA’s drug center for more than two decades before her 2024 retirement.
The dual-study requirement originated in the early 1960s when Congress enacted legislation mandating FDA review of data from “adequate and well-controlled investigations” before approving new treatments. For many years, agency officials interpreted this mandate as necessitating at least two comprehensive studies, typically involving large patient populations and extended monitoring periods.
The purpose behind requiring a second trial was to verify that initial study outcomes weren’t anomalous and could be replicated in different circumstances.
However, starting in the 1990s, FDA officials increasingly began accepting single studies for treatments targeting rare or deadly diseases, where companies frequently face challenges conducting large-scale patient trials.
During the past five years, approximately 60% of groundbreaking drugs approved annually have received clearance based on single studies. This trend reflects congressional legislation directing regulators toward greater flexibility when evaluating treatments for severe or difficult-to-treat medical conditions.
According to Woodcock, Wednesday’s policy announcement will primarily affect medications for common diseases that previously didn’t qualify for reduced testing requirements.
“It’s not the cancers and the rare diseases that will be affected by this,” she explained. “The agency has been approving those on a single trial already.”
The current FDA leadership’s strategy contrasts sharply with recent agency decisions regarding vaccines, gene therapies, and other treatments.
Last week, the FDA’s vaccine division, under Prasad’s direction, initially rejected Moderna’s application for a new mRNA influenza vaccine, citing inadequate clinical trial data. However, on Wednesday, the agency reversed its position and agreed to review the vaccine after Moderna committed to conducting additional studies involving elderly patients.
Additionally, Prasad has declined approval for numerous experimental gene therapies and biotechnology drugs, demanding additional research or more conclusive evidence. This pattern has negatively impacted biotech company stock values and contradicted Makary’s public statements promoting expedited and flexible FDA reviews.
Woodcock noted that pharmaceutical companies must wait to determine whether the FDA’s approach to promising experimental treatments will actually change.
“Implementation will be everything,” she said. “Since the agency’s approach is unclear, and the industry is already baffled, I don’t think this adds any illumination.”
