A new survey reveals that U.S. biotechnology companies focused on developing treatments for rare and serious diseases overwhelmingly prefer to conduct first-in-human (FIH) clinical trials in the United States. However, historical obstacles posed by the U.S. Food and Drug Administration (FDA) are prompting them to seek development overseas – in Australia, Europe, and China. The survey of 37 U.S. biotechnology executives was conducted by the Biotech Consortium to Accelerate Innovation (BCAI) in partnership with MassBio.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260812482557/en/
When the FDA review criteria are consistent, the U.S. emerges as the clear favorite for FIH trials, boasting a mean rank of 1.49 and being the top choice for 76% of companies surveyed. However, in the historical regulatory landscape, this dynamic changes: Australia surpasses the U.S. with a mean rank of 2.67 compared to 4.03. This transition is not indicative of the quality or location of the scientific research, but rather reflects the sponsors’ perception of obtaining a timely, reliable response from regulatory authorities.
Results from the survey show:
- 76% pick the U.S. as the #1 choice for FIH trials, when review is predictable.
- 72% are hesitant to run FIH trials in the U.S. under FDA’s historical framework.
- 73% say the top reason why sponsors are leaving the U.S. is delay and financial cost tied to FDA clinical holds and rework.
- Surveyed executives cite “current turmoil at the FDA with high turnover” and warn the U.S. is “offshoring our industry faster than we can catch up.” 54% say they are now less likely to test new drugs in the U.S.
- 85% support fixing FDA’s “1/10 rule” so it sets a safe starting dose, not a treatment ceiling.
Proposed solutions that can be implemented by the FDA, as identified from the survey:
- Clarify that the 10x safety margin sets a starting dose, not a treatment ceiling. Surveyed companies are not requesting the FDA to compromise on patient safety. Instead, they are asking the FDA to implement the 10x safety margin as outlined in the Agency’s own 2005 guidance, Estimating the Maximum Safe Starting Dose in Initial Clinical Trials1.
- Restore predictable review timelines that reduce costly holds and rework.
- Strengthen sponsor-reviewer communication so trial design is driven by science, not procedural ambiguity.
- Ensure better alignment of review criteria with disease severity and life expectancy.
- Improve benefit/risk framework which considers the patient voice.
- Utilize a central Institutional Review Board for first-in-patient studies.
Survey Methodology
Findings are based on a survey of 37 U.S. biotechnology executives conducted by BCAI in partnership with MassBio between December 2025 and March 2026. Most respondents are small, early stage organizations (76% with fewer than 50 employees) developing therapies in neurology/central nervous system, oncology, and immunology.
About the Biotech Consortium to Accelerate Innovation (BCAI)
The Biotech Consortium to Accelerate Innovation (BCAI) unites more than a dozen innovative biotechnology companies developing transformative therapies for serious neurological and rare diseases, including amyotrophic lateral sclerosis, frontotemporal dementia, Angelman syndrome, and Duchenne muscular dystrophy. BCAI’s mission is to keep American clinical trials in the U.S., so American patients have the earliest possible access to U.S.-developed medical innovation.
About MassBio
MassBio is the driving force behind Massachusetts’s life sciences ecosystem, supporting innovation and industry growth by offering best-in-class resources to over 1,700 member organizations at all stages of the biopharma lifecycle. Founded in 1985, MassBio aspires to extend Massachusetts’s impact as the global center of excellence in biomedical breakthroughs. Through strategic cost-saving initiatives, robust business partnerships, educational and networking opportunities, and proactive advocacy, MassBio empowers its members to launch the next generation of medical advancements to deliver the cures and therapies that enhance patient lives.
Reference
1U.S. Food and Drug Administration. Estimating the Maximum Safe Starting Dose in Initial Clinical Trials for Therapeutics in Adult Healthy Volunteers: Guidance for Industry. July 2005. Available at: https://www.fda.gov/media/72309/download
View source version on businesswire.com: https://www.businesswire.com/news/home/20260812482557/en/
Media gallery
