CAMBRIDGE, Mass. — U.S. biotechnology companies developing treatments for rare and serious diseases overwhelmingly prefer to conduct first-in-human clinical trials in the United States, but regulatory uncertainty is pushing many to consider trials in Australia, Europe and China, according to a new industry survey.
The survey of 37 U.S. biotechnology executives was conducted by the Biotech Consortium to Accelerate Innovation in partnership with MassBio.
When regulatory review is predictable and consistent, 76% of respondents ranked the United States as their top choice for first-in-human trials. However, 72% said they were hesitant to conduct such trials in the U.S. under the FDA’s historical regulatory framework.
Australia ranked ahead of the United States when respondents considered the historical regulatory environment, according to the survey. The organizations said the shift reflected concerns about obtaining timely and predictable responses from regulators rather than the quality of U.S. scientific research.
The survey found that 73% of respondents identified delays and financial costs associated with FDA clinical holds and additional work as the primary reason sponsors move trials outside the United States. Fifty-four percent said they are now less likely to test new drugs in the country.
Respondents also expressed concern about turnover and instability at the FDA, with some warning that regulatory uncertainty could accelerate the movement of biotechnology development overseas.
The survey found that 85% of respondents support changes to the FDA’s so-called “1/10 rule,” which companies said should be used to establish a safe starting dose rather than limit subsequent dose escalation.
Among other proposed changes, respondents called for more predictable FDA review timelines, stronger communication between sponsors and reviewers, greater alignment of review criteria with disease severity and life expectancy, broader consideration of patient perspectives in benefit-risk assessments and increased use of central Institutional Review Boards for first-in-patient studies.
The organizations emphasized that companies were not seeking weaker patient safety standards, but greater clarity around implementation of the FDA’s existing guidance on determining safe starting doses in initial clinical trials.
The survey was conducted between December 2025 and March 2026. Most respondents represented small, early-stage biotechnology companies, with 76% employing fewer than 50 people. Participating companies were primarily developing therapies in neurology and central nervous system disorders, oncology and immunology.
