FDA Launches Expedited IND Pilot to Speed First-in-Human Trials

The FDA has launched the Expedited Investigational New Drug (IND) Pilot Program and began accepting applications from drug sponsors and prospective partner institutions, with the stated goal of shortening the time from drug identification to first-in-human clinical trials.¹

The pilot aims to pair drug sponsors with qualified research institutions including academic medical centers, health networks, contract research organizations, or regulatory advisory firms that provide nonclinical, clinical, and chemistry, manufacturing, and controls (CMC) expertise during IND preparation. Because qualified research institutions are expected to bring substantial scientific expertise to that process, the FDA will accept and review individual IND components on a rolling basis rather than waiting for a complete submission package.^1,2

The agency is accepting applications from sponsor-institution pairs through October 30, 2026, and expects to select 8 to 10 pairs for the initial pilot cohort by December 18, 2026. Eligible programs must target a phase 1 first-in-human submission reviewed by the Center for Drug Evaluation and Research (CDER) Office of New Drugs, the Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products, or the Oncology Center of Excellence (OCE).

“The pilot not only pairs industry innovators with top research institutions to accelerate high-quality data being submitted to the FDA, [but] it also tests if the partnership can accelerate what happens after the FDA allows a clinical trial to proceed,” acting FDA commissioner Kyle Diamantas, JD, stated in a news release.

Program Design and Eligibility

According to the FDA, first-in-human trials currently take up to 2 years to complete in the US, compared with faster timelines in China and Australia.¹ Selection considerations for participating sponsors include the complexity of the IND and its fit with the rolling-review format, the development stage of the nonclinical and CMC program, public health impact and unmet medical need, and sponsor size and IND experience; the agency stated that applications representing rare diseases, pediatric indications, or novel modalities may contribute to cohort diversity.² Prospective research institutions are evaluated on documented expertise across nonclinical, CMC, clinical, clinical pharmacology, and regulatory affairs disciplines, along with either owned clinical trial infrastructure or documented site partnerships to support phase 1 activation. The FDA retains full regulatory authority throughout, including all clinical hold and safe-to-proceed determinations.

“The pilot hopes to utilize the American innovation ecosystem to accelerate the time to first-in-human clinical trials,” said Karim Mikhail, BPharm, MS, director of CBER, in the news release. “The FDA is doing our part to ensure American patients continue to receive access to therapies first, while keeping scientific innovation and investment in America.”¹

Background: Operation TrialBlazer and PDUFA Reauthorization

The pilot is a component of the Department of Health and Human Services’ Operation TrialBlazer, a department-wide initiative announced June 22, 2026, aimed at reversing the shift of early-stage clinical research overseas. Beyond the FDA pilot, Operation TrialBlazer includes National Institutes of Health (NIH) efforts to support well-powered trials using artificial intelligence, human cell-based models, and real-world data; work by NIH’s National Center for Advancing Translational Sciences on accelerating rare disease therapy development; and National Cancer Institute efforts to streamline cancer trial activation and enrollment.³

In a public meeting on September 16 regarding the reauthorization of the Prescription Drug User Fee Act (PDUFA), the FDA included a proposal that would cut new drug application fees by 50% for submissions that include data from at least 1 phase 1 trial conducted in the US after October 1, 2027.⁴

“The FDA greatly appreciates the public feedback we received on the proposed pilot, and we have incorporated that feedback into the final pilot design,” said Michael Davis, MD, PhD, director of CDER, in the news release. “The FDA is committed to ensuring the United States remains the global standard for pharmaceutical innovation and regulatory rigor for the benefit of American patients and innovators.”

REFERENCES

1. FDA Launches Expedited IND Pilot, Begins Accepting Applications. News release. U.S. Food and Drug Administration. September 15, 2026. Accessed September 17, 2026. https://tinyurl.com/mr3m4p8x

2. FDA Expedited Investigational New Drug (IND) Pilot Program: Accelerating Early-Stage Clinical Research in the U.S. U.S. Food and Drug Administration. Accessed September 17, 2026. https://tinyurl.com/yxby5a6z

3. HHS Launches Unprecedented Department-Wide Effort to Restore American Leadership in Clinical Trials. News release. U.S. Department of Health and Human Services. June 22, 2026. Accessed September 17, 2026. https://tinyurl.com/fn7xk2d4

4. Food and Drug Administration, HHS. Reauthorization of the Prescription Drug User Fee Act; public meeting; request for comments. Fed Regist. 2026;91(156):52702-52708. Accessed September 17, 2026. https://tinyurl.com/5bk7bwyt