Major clinical trial–related policy changes at the U.S. Department of Health and Human Services (HHS) are leading to ambiguity for sites, sponsors, and contract research organizations. Questions remain on how clinical research professionals can best operationalize these changes, align processes, and address compliance challenges.
One important change is the U.S. Food and Drug Administration’s (FDA’s) plan to require one pivotal clinical trial to consider a drug for approval, instead of the traditional two trials. “In principle, this should reduce development costs and timelines, but questions remain as to who makes the decision on which drugs will still require two studies,” notes Catherine Gregor, MBA, CCRP, CCRC, Chief Clinical Trial Officer at Florence Healthcare. “There are widespread concerns about an apparent lack of scientific rigor in FDA decision-making.”
“Frequent changes in FDA leadership as well as high turnover in experienced agency staff have increased anxiety levels among clinical research professionals,” notes Sophia McLeod, Advocacy Advisor at the Association of Clinical Research Organizations (ACRO). “This adds a level of unpredictability to interactions with the agency. Also, there is a now a lack of formal processes for stakeholders to provide input on proposed policy changes.”
McLeod explains that there are competing priorities in terms of guidance on artificial intelligence (AI), with draft FDA guidance for industry on use of AI in regulatory decision-making, various legislation at the state level, and working groups on the Hill debating what a federal legislative package should contain. Added to this is an Executive Order issued on December 11, 2025, aimed at driving U.S. leadership in AI by ensuring that there is a “minimally burdensome national standard—not 50 discordant State ones.”
Trials in Transition: Navigating HHS Policy Shifts in 2025
Join Catherine and Sophia at ACRP 2026 [April 24-27; Orlando, Fla.] as they explore five major developments from the recent HHS policy changes impacting how clinical trials are conducted in the U.S. View complete schedule.
FDA itself is increasingly using AI, including a large language model–based tool, Elsa, and agentic AI. The agency expects complex tasks such as “pre-market reviews, review validation, post-market surveillance, inspections, and compliance and administrative functions” to benefit from agentic AI.
“While AI is intended to make agency review processes more efficient, vendors navigating this environment face challenges due to the fact that sponsors and sites are highly risk averse,” says Gregor. “We’re hearing sponsors say that ‘it’s OK to use AI for the icing, but not for the cake.’”
Edited by Jill Dawson
