The U.S. Food and Drug Administration is preparing to introduce a significant policy shift that may streamline the pathway for new drugs and medical products. According to comments made to STAT by FDA Commissioner Marty Makary, the agency plans to establish a new default standard requiring one pivotal clinical study, rather than the longstanding expectation of two, before an application is considered for approval.
A Change in Regulatory Paradigms
Historically, the FDA has relied on two adequate and well-controlled trials as the benchmark for demonstrating a product’s safety and efficacy. This approach provided confirmatory evidence and reduced uncertainty in regulatory decision-making. Over the past decade, however, the agency has demonstrated increasing flexibility, especially through accelerated approval pathways, orphan drug programs, and situations involving high unmet medical need.
Many developers already submit a single pivotal clinical study, supplemented by supportive data, real-world evidence, or secondary analyses. The new policy seeks to formalize what has gradually become common practice.
Details of the New Default Standard
Commissioner Makary emphasized that while the default will now be one pivotal trial, the FDA will not eliminate the option—or the requirement of a second study where warranted. Products with complex mechanisms, inconsistent early-stage data, narrow therapeutic windows, or higher safety risks may still necessitate more extensive evidence packages.
This approach aims to introduce efficiency without compromising scientific rigor. The FDA remains responsible for ensuring that evidence supporting approval is robust, reproducible, and reliable.
Implications for Drugmakers and Patients
For industry stakeholders, the policy change could lead to reduced development timelines, lower research costs, and earlier submission opportunities. It may also encourage smaller companies and innovators working in niche therapeutic spaces by lowering the evidentiary threshold for entry into the regulatory process.
For patients, particularly those with rare or serious diseases, this shift may facilitate faster access to new therapies. At the same time, the FDA maintains its oversight mechanisms to monitor post-marketing safety and confirm long-term outcomes.
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Written by Nare Hovhannisyan, MD