Last month, the FDA launched TrialBlazer, intended to streamline the IND path and bring early clinical trials and medical innovation home to the U.S. It’s a start, but new agency leadership must see it through.
I can’t help but think: the FDA was built for a different time.
I recently sat in a room of 10 biotech executives, and none are doing first-in-human clinical trials in the U.S. That should stop us in our tracks.
As CEO and president of MassBio, I have watched this shift firsthand. Not long ago, running a first-in-human trial in the United States was simply the default. It did not require a decision or justification. For a growing number of biotechs, that is no longer true.
One of the main drivers of this change is that the FDA was built for a different era and type of drug development. Created more than 120 years ago, the contemporary FDA’s frameworks reflect a deep understanding of how large, established companies operate but an insufficient appreciation for the realities of early-stage drug development. Over the past few decades, much of the industry’s early-stage medical innovation has come from leaner biotechs, where small teams must make high-stakes decisions with limited capital and narrow runways. The FDA has not yet modernized to this reality.
To be clear, this is not a Big Pharma-versus-biotech issue. But the regulatory system must work equally well for companies of all sizes. That is the only way scientific and medical innovation will continue to advance, reach patients faster and, importantly, stay here in the U.S. as one of the country’s most important economic engines.
With vacancies at the agency across many critical positions, including FDA commissioner and director of the Center for Biologics Evaluation and Research (CBER), it is imperative for new agency leadership to prioritize setting up a system that ultimately operates better for everyone—pharma, biotech and patients.
To achieve this, the FDA’s next chapter must evolve specifically with biotech in mind. It must prioritize transparency, clear guidance and regulatory processes that reflect how drug development happens today. Patients cannot afford a system where promising therapies are slowed by avoidable ambiguity—where sponsors cannot tell what the agency truly requires versus what it merely prefers—and emerging biotech companies cannot succeed in an environment where unpredictability impacts whether science advances.
We know this is possible because other countries—the ones where those 10 companies are running early clinical trials—are already operating with smaller companies in mind.
The FDA has begun to move. The agency now shares its reasoning publicly when it rejects an investigational drug, and the FDA and the Department of Health and Human Services (HHS) recently launched Operation TrialBlazer, a roadmap aimed at keeping early clinical research in the U.S. through clearer Phase 1 requirements, a streamlined pre–Investigational New Drug (IND) path and new sponsor resources. The diagnosis is right, and it echoes what biotech leaders have been saying for years. But a roadmap launched under acting leadership only matters if it is implemented with industry input and built to last, and biotechs still need clearer insight into how decisions are made and how expectations evolve.
TrialBlazer’s new Phase 1 contact center and expedited-IND pilot point in this direction, but a help line does not go far enough. It is not a dedicated emerging-biotech function inside the FDA, empowered to resolve misalignments before they become costly delays, and a pilot is not yet permanent policy. There needs to be a commitment to real-time meetings, not just written feedback, paired with predictable review timelines, so that an early-stage company can map its path to first-in-human trials here in the U.S. with the confidence it now finds overseas.
In a recent MassBio survey of biotech executives, 55% said the FDA’s written feedback was ambiguous or confusing. When biotech companies believe they are aligned with FDA feedback only to encounter different expectations later in review, the consequences are real.
Today’s biotech landscape is innovation-forward but highly dependent on early validation points. Investors can tolerate scientific risk but not uncertainty and unclear direction in the regulatory process. In the same survey, respondents reported that 78% of FDA meeting requests over the past five years resulted only in written responses, while far fewer included face-to-face engagement with meeting minutes. Written feedback has a role, but it cannot replace real-time dialogue where questions can be asked, nuance can be explained and misinterpretations can be corrected before they affect development plans, sometimes adding months or years to timelines.
Countries like China, Australia, the United Kingdom, Canada and the Netherlands are increasingly seen as more predictable environments for early-stage clinical development, offering clearer communication, faster feedback and a more collaborative posture. China’s early timelines run an estimated 50% to 70% faster than the rest of the world, a figure cited in the FDA’s own TrialBlazer roadmap, and Australia has become a major destination for early-stage trials for the same reasons.
When biotech companies in our earlier survey were asked whether they would choose China for early development if the playing field were level with the United States, zero said they would. U.S. science has not lost its advantage, and companies are not eager to leave. The primary variable pushing companies abroad is the regulatory experience, and this is a problem fully within our power to solve. Get the regulatory environment right by delivering the speed, clarity and engagement of foreign systems, and the strongest reason to look anywhere but here disappears.
Whoever takes the helm at the FDA permanently will inherit TrialBlazer and a rare opportunity to define the agency’s next chapter, ensuring these reforms outlast this transition and determining whether the United States remains the place where the world’s most promising medicines are born.
