FDA lowers drug approval standards: Now accepting single clinical trials

02/24/2026 / By Belle Carter

The FDA now allows many new drugs to be approved based on just one clinical trial, abandoning its long-standing requirement of two studies for most approvals. This change reflects evolving scientific understanding but raises concerns about reduced evidentiary standards.
Since the 1960s, the FDA typically required two well-controlled trials to confirm safety and efficacy. However, since the 1990s, exceptions for rare diseases and terminal conditions have increased, with 60% of recent novel drugs approved via single trials.
Proponents argue modern biology justifies fewer trials, while critics warn that relying on single studies leaves patients unsure of real-world benefits. Experts like Dr. Reshma Ramachandran question whether this lowers standards rather than improving them.
The FDA plans to offset risks by enhancing post-approval monitoring and retaining authority to demand additional studies if needed. However, skeptics doubt whether this will adequately protect patients from insufficiently tested drugs.
The policy aims to accelerate drug development and reduce costs, but its long-term impact remains uncertain. The effectiveness of postmarket oversight will determine whether faster approvals compromise patient safety.

The U.S. Food and Drug Administration (FDA) will now approve many new drugs based on a single clinical trial—a major departure from its long-standing requirement of two studies for most approvals.

The shift, announced by FDA Commissioner Dr. Marty Makary and Dr. Vinay Prasad of the Center for Biologics Evaluation and Research, reflects the agency’s evolving approach to drug evaluation in an era of advanced biological understanding.

According to BrightU.AI‘s Enoch, the FDA has historically relied on at least two well-controlled studies to confirm a drug’s safety and effectiveness. But in recent years, the agency has increasingly accepted single-trial approvals, particularly for treatments targeting rare or life-threatening conditions. Now, the FDA is formalizing this flexibility, making a single well-conducted trial—backed by confirmatory evidence—the new default standard.

A decades-old standard gives way

The two-study benchmark traces back to the early 1960s, when Congress mandated that the FDA evaluate data from “adequate and well-controlled investigations” before approving new medications. For decades, this meant at least two trials, often involving large patient groups and extended follow-up periods. The second study served as a safeguard, ensuring initial results were reproducible and not due to chance.

However, in the 1990s, the FDA began accepting single trials more frequently, especially for drugs treating rare or terminal illnesses where recruiting large numbers of patients was impractical. Over the past five years, roughly 60% of first-of-a-kind drugs have been approved based on a single study.

Makary and Prasad argue that advances in biomedical science have rendered the two-trial requirement outdated.

“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,” they wrote in the New England Journal of Medicine.

Supporters of the change, including former FDA drug center director Dr. Janet Woodcock, say it aligns with scientific progress.

“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.

But critics warn that reducing trial requirements could leave patients with lingering uncertainties about drug effectiveness. Dr. Reshma Ramachandran of Yale School of Medicine noted on X that while most recent approvals have relied on single trials, patients often remain unsure of a drug’s real-world benefits.

“Why set a standard continuing the (bad) same old instead of demanding more?” she questioned.

Postmarket surveillance as a counterbalance

To mitigate risks, the FDA plans to pair the new single-trial policy with enhanced postmarket monitoring. Makary and Prasad emphasized that the agency retains authority to demand additional studies if trial data prove insufficient.

“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 shift is expected to accelerate drug development and reduce costs, potentially encouraging innovation—but whether it strikes the right balance between speed and safety remains to be seen.

The FDA’s move signals a broader trend toward streamlining drug approvals amid rapid scientific advancements. While proponents argue that modern biology justifies fewer trials, skeptics caution that weaker evidentiary standards could expose patients to greater uncertainty. As the policy rolls out, the effectiveness of postmarket surveillance will be critical in ensuring that faster approvals don’t come at the expense of patient safety.

For now, the FDA’s pivot marks a decisive step away from tradition—one that could reshape the pharmaceutical landscape for years to come.

Watch the video below that talks about the FDA demanding real vaccine trials.

This video is from The HighWire with Del Bigtree channel on Brighteon.com.