Agency leaders say one well-designed study will become the default requirement, paired with expanded postmarket data collection.
By yourNEWS Media Newsroom
The Food and Drug Administration will move toward approving many new drugs based on a single clinical trial, agency leaders said, marking a significant change from the long-standing expectation of two studies for most approvals.
According to statements reported by The Epoch Times, FDA officials said the agency’s new default standard will require one well-conducted trial, accompanied by confirmatory evidence and enhanced postmarket monitoring.
“The FDA has demonstrated disease-by-disease flexibility and has granted approvals based on a single premarket study with confirmatory evidence. In some fields, such as oncology, single trials have supported the majority of drug approvals,” FDA Commissioner Dr. Marty Makary and Dr. Vinay Prasad, head of the FDA’s Center for Biologics Evaluation and Research, wrote in a Feb. 18 article published in the New England Journal of Medicine.
“However, although we have exercised flexibility in the past, there remains confusion from manufacturers regarding settings in which a single trial will be accepted. Moving forward, we are announcing that a one-trial requirement will be the FDA’s new default standard. This reform is being rolled out synchronously with the agency’s postmarket initiative to collect robust data on all drugs and devices.”
The two-study benchmark dates to the early 1960s, when Congress required the FDA to evaluate data from “adequate and well-controlled investigations” before approving new medications. For decades, the agency interpreted that to mean at least two studies, typically involving substantial patient enrollment and follow-up.
The rationale for a second trial was to confirm that the findings of the first were reproducible and not the result of chance.
Beginning in the 1990s, the FDA began accepting single studies more frequently, particularly for treatments targeting rare or life-threatening conditions where conducting multiple large trials proved difficult. Over the past five years, approximately 60 percent of first-of-a-kind drugs have been approved on the basis of a single study.
Makary and Prasad wrote that reliance on multiple trials “was intended to provide credible causal evidence that a therapy could improve clinical outcomes with acceptable safety in a world where biologic understanding was more limited than it is today.”
They added, “In the modern world, as drug discovery becomes increasingly precise and scientific, the FDA considers not just effects on survival, but biochemical and intermediate changes that tell a complete biologic story: does this drug actually work? In this setting, overreliance on two trials no longer makes sense.”
Agency leaders said the shift is expected to reduce development costs and shorten the time required to bring drugs to market. They anticipate that the change could encourage additional drug development.
Dr. Janet Woodcock, who led the FDA’s drug center for roughly two decades before retiring in 2024, said the move aligns with a broader trend the agency has followed for years, particularly for life-threatening diseases such as 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,” Woodcock said.
Not all experts expressed support. Dr. Reshma Ramachandran, an assistant professor of medicine at the Yale School of Medicine, wrote on X that while most recent FDA approvals have relied on single strong trials, concerns remain.
“But as the authors noted (& have for years!), patients are increasingly left with uncertainty of their effectiveness,” she wrote, “so why set a standard continuing the (bad) same old instead of demanding more?”
Makary and Prasad emphasized that the agency retains authority to require additional studies when necessary.
“Instead of prioritizing finite reviewer time reading and assessing two or more pivotal trials, we will focus our energies on ensuring that the one clinical trial we require provides the most up-to-date and useful information for American patients,” they wrote.
The policy change is expected to be implemented alongside expanded postmarket surveillance intended to collect ongoing data on approved drugs and medical devices.
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