Survey Finds US Biotechs Prefer Domestic First-in-Human Trials but FDA Hurdles Are Pushing Them Abroad

United States biotechnology companies developing treatments for rare and serious diseases overwhelmingly prefer to run first-in-human (FIH) trials in the US. However, historical obstacles at the FDA are pushing them toward Australia, Europe, and China instead. That is the central finding of a new survey of 37 US biotech executives conducted by the Biotech Consortium to Accelerate Innovation (BCAI) in partnership with MassBio.¹

When FDA review criteria are consistent and predictable, the US emerges as the clear favorite, ranking first for 76% of companies surveyed with a mean rank of 1.49. But under the historical regulatory landscape, that dynamic shifts: Australia surpasses the US, with a mean rank of 2.67 compared to 4.03 for the US. The survey notes this shift does not reflect the quality of US science, but rather sponsors' perception of obtaining timely, reliable responses from regulators.

Seventy-two percent of respondents said they are hesitant to run FIH trials in the US under FDA's historical framework, and 73% identified delays and financial costs tied to FDA clinical holds and rework as the top reason sponsors are leaving.

Fifty-four percent said they are now less likely to test new drugs in the US, with surveyed executives citing "current turmoil at the FDA with high turnover" and warning that the country is "offshoring our industry faster than we can catch up."

What sponsors say needs to change

The survey identified several proposed solutions. Eighty-five percent of respondents support fixing the FDA's "1/10 rule" so that it functions as a safe starting dose, not a treatment ceiling. The survey clarifies that respondents are not asking the FDA to compromise on patient safety, but rather to implement the 10x safety margin as outlined in the agency's own 2005 guidance on estimating the maximum safe starting dose in initial clinical trials.

Additional proposals include restoring predictable review timelines to reduce costly holds and rework, strengthening sponsor-reviewer communication so that trial design is driven by science rather than procedural ambiguity, improving benefit-risk frameworks to incorporate the patient voice, and utilizing a central institutional review board for first-in-patient studies.

Most respondents represent small, early-stage organizations, with 76% employing fewer than 50 people. Therapeutic focus areas include neurology and central nervous system conditions, oncology, and immunology.

Broader pressures on early-stage development

The survey findings reflect a broader set of pressures reshaping how early-stage biotechs approach clinical development. In a recent video interview with Applied Clinical Trials, Stella K. Vnook, PhD, chief executive officer and executive chair of Kaida BioPharma and founder and chief executive officer of Aviva Ventures, described a trifecta of converging forces affecting the field.²

"There are significant changes in NIH funding, which has traditionally funded early clinical programs," Vnook said. "There's also a cliff in large pharma—they're seeking assets, but later-stage assets. And third, the global ability to get a clinical trial done and get that proof-of-concept human data has really motivated all of us to rethink our development plans, our regulatory strategy, and our patient population."

The result, she noted, is a field that has fundamentally shifted its priorities. "Maybe four years ago, we were much more interested in very creative science and interesting approaches. Now we're focused on going really fast, utilizing less capital to get to inflection points, and getting to first patient in as soon as we can."

On risk, Vnook was clear about where the most consequential threat now lies. "The most significant risk is not seeing the potential of real assets because we don't have enough funding to get there," she said.

This is a concern that maps closely onto what the BCAI survey captures: a biotech community that knows where it wants to run trials, but is being forced to recalculate based on regulatory and financial realities.

References

1. New Survey Finds U.S. Biotechs Advancing Treatments for Rare and Serious Diseases Want to Run Clinical Trials in U.S. but Regulatory Hurdles are Sending Them Abroad. News release. MassBio. Biotech Consortium to Accelerate Innovation. August 12, 2026. Accessed August 13, 2026. https://www.businesswire.com/news/home/20260812482557/en/New-Survey-Finds-U.S.-Biotechs-Advancing-Treatments-for-Rare-and-Serious-Diseases-Want-to-Run-Clinical-Trials-in-U.S.-but-Regulatory-Hurdles-are-Sending-Them-Abroad
2. How the Funding Trifecta Is Reshaping Clinical Development Priorities. Applied Clinical Trials. August 4, 2026. Accessed August 13, 2026. https://www.appliedclinicaltrialsonline.com/view/funding-reshaping-clinical-development-priorities