Why America Is Losing the Clinical Trial Race
The United States is no longer the undisputed leader in clinical research. That shift is now a federal concern.
The Department of Health and Human Services launched a department-wide initiative this week to accelerate domestic clinical research. Federal health officials want to restore the country’s edge in a fast-changing global competition for biomedical innovation. The effort spans multiple agencies — the FDA, NIH, ARPA-H, and ONC — all working toward one goal: bring early-stage drug development back to American soil.
The China Factor
The numbers behind this push are striking. China surpassed the United States in total registered clinical trials in 2024. Chinese institutions accounted for more than 7,100 trials, or roughly 39% of all global clinical studies. Moreover, China exceeded the U.S. in Phase 1 clinical trial activity for the first time back in 2021. That trend has continued to widen.
Federal officials say lengthy timelines, regulatory uncertainty, and rising costs have pushed pharmaceutical companies to conduct more early-stage research abroad. Countries like China have expanded clinical research infrastructure, increased patient recruitment capacity, and shortened study startup times. The U.S. risks being left behind.
What Operation Trailblazer Actually Proposes
The HHS roadmap — called Operation Trailblazer — outlines specific goals: shorten drug development timelines, expand domestic clinical trial activity, and increase patient access to experimental therapies.
Health Secretary Robert F. Kennedy Jr. stated plainly that the current system has failed. “America should be the best place in the world to develop new medicines, yet we have built a system that drives too much clinical research overseas,” he said. Under President Trump’s direction, Kennedy added, HHS will work to remove unnecessary barriers and bring investment back home.
A Coordinated Multi-Agency Effort
Rather than a single agency acting alone, Operation Trailblazer coordinates action across four key federal health bodies. Each plays a distinct role:
- FDA — streamlining early-stage trial approvals and regulatory clarity
- NIH — expanding support for large, AI-assisted clinical studies
- ARPA-H — advancing precision medicine and computational drug analysis programs
- ONC — integrating clinical trial data into electronic health records
This kind of coordinated approach is new. Historically, these agencies have operated in parallel rather than together.
FDA’s New Fast-Track Rules for Drug Development
At the center of HHS’s plan sits a sweeping FDA proposal. The agency wants to cut the time needed to move experimental therapies from laboratory discovery into first-in-human clinical trials.
Expedited IND Pilot Program
The FDA opened a public comment period on a new Expedited Investigational New Drug (IND) pilot program. Under this proposal, qualified academic medical centers, contract research organizations, and other institutions can work directly with drug sponsors to develop Phase 1 clinical trial protocols. Participants would use a rolling submission process that reduces regulatory delays. It also lowers the risk of clinical holds before studies even begin. Comments are open through July 22.
Single-Trial Approval Pathway
Additionally, the FDA issued draft guidance on a significant policy shift. The agency clarified when a single, well-conducted late-stage clinical trial — combined with strong confirmatory evidence — may be enough to support drug or biologic approval. This changes the traditional FDA expectation of two adequate and well-controlled trials. Officials say it offers greater regulatory clarity without sacrificing scientific standards.
QSP Models for Dosing Decisions
A separate draft guidance addresses how quantitative systems pharmacology (QSP) models can determine safe starting doses for first-in-human studies. These advanced computational models simulate biological systems and predict drug responses before any human testing begins. The FDA sees this as part of a broader push to reduce reliance on animal toxicology studies when evaluating complex therapies.
How NIH and ARPA-H Are Stepping Up
NIH is expanding support for large, well-powered clinical studies. The agency is also increasing its use of artificial intelligence, real-world evidence, and advanced human cell-based research models.
NCATS and Translational Research
NIH’s National Center for Advancing Translational Sciences (NCATS) is building on research that contributed to the world’s first personalized CRISPR-based gene-editing treatment. Meanwhile, the National Cancer Institute is partnering with cancer centers to streamline study activation and improve patient enrollment rates.
ARPA-H’s Precision Programs
ARPA-H is advancing two programs with strong implications for drug safety and rare disease treatment:
- THRIVE (Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines) — focuses on genetic therapies for rare conditions
- CATALYST (Computational ADME-Tox and Physiology Analysis for Safer Therapeutics) — uses AI and computational methods to evaluate drug behavior and safety earlier in development
Both programs aim to catch safety issues before they reach patients — not after.
The Role of AI and Computational Models
Artificial intelligence runs through almost every component of this initiative. From NIH’s use of AI in large-scale studies to ARPA-H’s computational drug safety programs, AI is no longer a supporting tool. It is a core strategy.
EHR Integration for Trial Enrollment
The Office of the National Coordinator for Health Information Technology (ONC) is exploring how to embed clinical trial opportunities directly into electronic health record (EHR) systems. Under this approach, eligible patients could learn about relevant research studies during routine medical appointments. This could dramatically expand the pool of trial participants by removing barriers to awareness and access.
The logic is simple: patients can’t enroll in trials they don’t know exist. Connecting EHRs to trial registries could change that.
Industry Voices: Support With Caution
Broad industry support exists for this initiative, but it comes with clear conditions.
Former FDA Commissioner Scott Gottlieb warned that the U.S. risks losing its leadership position if clinical development continues migrating overseas. At the same time, he cautioned that other nations have no inherent scientific advantage. The U.S. still holds significant structural strengths — its venture capital ecosystem, startup culture, and academic research networks remain the envy of the world.
Industry analysts echo that view. They note that China and other countries have invested heavily in trial infrastructure and patient recruitment. Consequently, the U.S. must act — but carefully. Regulatory modernization must not come at the cost of scientific rigor.
What Comes Next
Several public comment periods remain open through July and August 2026. After reviewing stakeholder feedback, federal agencies will decide whether proposals move into formal pilot programs, final guidance documents, or broader regulatory reforms.
In addition, HHS’s Office of Inspector General issued a request for information. It asks whether changes to anti-kickback safe harbor regulations or beneficiary inducement rules could help more patients participate in clinical trials.
The stakes are high. Clinical research drives drug discovery. Drug discovery drives patient outcomes. And right now, too much of that work is happening outside the United States.
If Operation Trailblazer delivers on its goals, the next wave of medical breakthroughs may once again begin on American soil.
