FDA Policy Shift: Single Study for New Drug Approvals
The Food and Drug Administration (FDA) plans to discontinue its long-standing requirement of two rigorous studies for new drug approvals. This significant policy change, announced by FDA Commissioner Dr. Marty Makary and Dr. Vinay Prasad, a top deputy, aims to accelerate the availability of specific medical products. The FDA's "default position" will now shift to requiring one study for new drugs and novel health products, as outlined in a New England Journal of Medicine piece published Wednesday.
This move marks another step by Makary and his team to alter long-standing FDA standards and procedures, aligning with their stated objective of reducing bureaucracy and expediting the availability of new medicines. Since April, Makary has implemented several directives, including mandating artificial intelligence use by staff and offering one-month drug assessments for new medications deemed to serve "national interests." This approach contrasts with the FDA's more restrictive stance on other products, such as vaccines.
The Rationale for Change
Makary and Prasad indicated that the decision to eliminate the two-trial requirement is based on modern advancements that have made drug research "increasingly precise and scientific."
"Overreliance on two trials no longer makes sense in the current research environment. 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 FDA officials predicted this policy shift would result in "a surge in drug development."
Expert Commentary and Historical Context
Dr. Janet Woodcock, former FDA drug director, commented that the change is logical and aligns with the FDA's historical trend of accepting one trial, supported by additional evidence, for certain life-threatening diseases like cancer. Woodcock, who headed the FDA's drug center for approximately 20 years before retiring in 2024, stated:
"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."
The two-study standard for drugs originated in the early 1960s, following a congressional law that mandated the FDA review data from "adequate and well-controlled investigations" prior to approving new medications. The agency historically interpreted this as requiring at least two studies, typically involving a large patient cohort and substantial follow-up. The rationale for a second study was to corroborate the initial trial's results and ensure reproducibility.
However, starting in the 1990s, the FDA began to increasingly approve treatments for rare or fatal diseases based on single studies, often due to challenges in testing such conditions in large patient populations. In the past five years, approximately 60% of first-of-a-kind drugs approved annually have received clearance based on a single study. This trend aligns with congressional directives for regulators to adopt greater flexibility in reviewing drugs for serious or difficult-to-treat conditions.
Woodcock indicated that the new policy will primarily affect drugs for common diseases that were not previously eligible for reduced testing standards.
"It’s not the cancers and the rare diseases that will be affected by this. The agency has been approving those on a single trial already."
Contrasting Approaches
This recent policy from FDA leadership contrasts sharply with the agency's concurrent actions regarding vaccines, gene therapies, and other treatments. Last week, the FDA’s vaccine division, led by Prasad, initially declined Moderna’s application for a new mRNA flu shot, citing insufficient clinical trial data. The agency later agreed to review the vaccine after Moderna committed to an additional study in older individuals.
Prasad has also rejected several experimental gene therapies and biotech drugs, citing requirements for further studies or more conclusive evidence. This trend has impacted the stock performance of numerous biotech companies and has been noted in relation to Makary's public statements on the speed and flexibility of FDA reviews.
Woodcock concluded that the drug industry awaits further clarity on whether the FDA's approach to promising experimental therapies will change.
"Implementation will be everything. Since the agency’s approach is unclear, and the industry is already baffled, I don’t think this adds any illumination."