The Food and Drug Administration is preparing to abandon its long-standing requirement that most new drugs be backed by two rigorous clinical trials, a move Trump-era appointees say is aimed at cutting red tape and speeding lifesaving treatments to patients.
According to Western Journal, the shift was outlined by FDA Commissioner Dr. Marty Makary and his top deputy, Dr. Vinay Prasad, in a commentary published Wednesday in The New England Journal of Medicine. Going forward, they wrote, the agencys default position will be to rely on a single pivotal study for new drugs and other novel medical products, supplemented by additional supporting evidence rather than a second full-scale trial.
The policy marks the latest in a series of reforms under Makary, who took over the agency last April pledging to streamline reviews and challenge entrenched bureaucratic habits that often slow innovation. Since his arrival, he has ordered staff to incorporate artificial intelligence into their work and has introduced one?month review timelines for drugs deemed to serve national interests.
Those steps underscore a broader philosophical shift away from the reflexive caution that has long characterized the FDA, particularly under Democratic administrations that tend to favor more regulation and slower approval pathways. It also highlights a growing divide within the agency itself, where some divisions are embracing flexibility while others cling to more restrictive standards, especially on politically sensitive products such as vaccines.
In their New England Journal article, Makary and Prasad argued that the two?trial rule is a relic of an earlier era and no longer reflects the sophistication of modern biomedical science. They wrote that advances in genetics, biomarkers, and trial design have made drug development increasingly precise and scientific, reducing the need to demand duplicate studies simply to confirm what one well?designed trial can already show.
In this setting, overreliance on two trials no longer makes sense, they wrote, adding that 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. The two officials predicted that the change would unleash a surge in drug development, particularly in areas where large, repetitive trials add cost and delay without significantly improving safety or effectiveness.
Support for the shift is coming from inside the FDAs own ranks, including from veterans who served under more regulation?friendly leadership. Dr. Janet Woodcock, the agencys former drug chief who ran the FDAs drug center for about two decades before retiring in 2024, said the move is consistent with a long?term trend toward greater flexibility in serious and life?threatening diseases.
The scientific point is well taken that as we move toward greater understanding of biology and disease we dont need to do two trials all the time, Woodcock said, noting that the agency has already been relying on single pivotal studies, plus corroborating data, for many cancer drugs and treatments for rare, often fatal conditions. Her comments underscore a key conservative critique of the regulatory state: once?sensible rules can calcify into rigid mandates long after science and technology have moved on.
The two?study standard dates back to the early 1960s, when Congress, reacting to drug safety scandals, required the FDA to base approvals on adequate and well?controlled investigations. For decades, regulators interpreted that phrase to mean at least two large, randomized trials with extensive follow?up, a framework that often favored big pharmaceutical companies able to absorb the cost and time of duplicative research.
The rationale for the second trial was straightforward: it served as a safeguard against fluke results and ensured that findings could be reproduced in a separate patient population. But as medicine has become more targeted and data?rich, critics have argued that insisting on a second full?scale trial in every case can delay access to promising therapies, drive up prices, and stifle competitionoutcomes that run counter to free?market principles and patient choice.
Beginning in the 1990s, the FDA quietly began to relax the two?trial norm for rare diseases and deadly conditions where large studies are difficult or impossible. Over the last five years, roughly 60 percent of first?in?class drugs have been approved on the basis of a single pivotal study, reflecting congressional directives that regulators show more flexibility when dealing with serious or hard?to?treat illnesses.
Woodcock emphasized that the newly announced policy will be most consequential for common conditions that previously did not qualify for streamlined standards. Its 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.
Even as Makary pushes to loosen some requirements, the FDA has taken a tougher line in other high?profile areas, particularly vaccines and cutting?edge biologics. Last week, the agencys vaccine division, led by Prasad, refused to accept Modernas application for a new mRNA flu shot, arguing that the companys clinical trial data were insufficient to justify review.
That hard?line stance triggered immediate pushback from industry and investors, but the FDA reversed course just days later, announcing it would consider the application after Moderna agreed to conduct an additional study in older adults. The episode illustrates the tension between public demands for rapid access to new technologies and the political and legal pressures on regulators to avoid any missteps, especially after the controversies surrounding COVID?19 vaccines and mandates.
Separately, Prasad has rejected a series of experimental gene therapies and biotech drugs, insisting on more definitive evidence or additional trials before granting approval. Those decisions have weighed on biotech stocks and have sometimes appeared at odds with Makarys public messaging about speed and flexibility, raising questions about how consistently the new one?trial default position will be applied across the agency.
Woodcock cautioned that the practical impact of the policy will depend less on headlines and more on how individual review divisions interpret their new leeway. Implementation will be everything, she said. Since the agencys approach is unclear, and the industry is already baffled, I dont think this adds any illumination.
For conservatives who have long argued that unelected regulators wield too much power over markets, innovation, and patient choice, the FDAs move away from a rigid two?trial mandate represents a modest but meaningful step toward a more flexible, evidence?driven framework. Whether that promise is realized will hinge on whether Makarys reforms translate into faster approvals for safe, effective drugs without the kind of arbitrary reversals and mixed signals that have plagued recent vaccine and gene?therapy decisions.
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