FDA Launches Pilot to Help Speed First Human Studies

Rare Daily Staff

The U.S. Food and Drug Administration has opened applications for a new pilot program intended to help drug developers move experimental medicines into first-in-human clinical testing more quickly.

The FDA’s Expedited Investigational New Drug, or IND, Pilot will pair selected drug sponsors with qualified research institutions that can help prepare the scientific and regulatory materials needed to begin a clinical trial in people. Applications are due October 30, 2026, and the agency expects to select eight to 10 sponsor–research institution pairs for the initial cohort.

“The pilot not only pairs industry innovators with top research institutions to accelerate high-quality data being submitted to the FDA, it also tests if the partnership can accelerate what happens after the FDA allows a clinical trial to proceed,” said Acting FDA Commissioner Kyle Diamantas.

For people living with rare diseases and their families, the announcement does not create a new approval pathway or guarantee that an experimental treatment will reach the clinic faster. But it could make the earliest stage of drug development more organized and predictable, especially for small biotechnology companies developing complex therapies.

Before a company can begin testing a new drug, biologic, gene therapy, or other product in people in the United States, it generally must submit an IND application to the FDA. The application includes extensive information about how the product is made, what laboratory and animal studies have shown, the dose researchers plan to use, and how the first clinical trial will protect participants.

The FDA then has 30 days to review the IND before the study can proceed, unless the agency places the trial on clinical hold because of unresolved safety or scientific concerns.

That formal 30-day period is only one part of the process. Preparing an IND-ready development package can take months or years. The FDA said first-in-human clinical trials may take as long as two years to initiate in the United States, and that the new pilot is intended to reduce avoidable delays between identifying a potential drug and starting the first study in people.

Under the Expedited IND Pilot, participating companies will work with qualified research institutions that have scientific and regulatory expertise relevant to preparing IND applications. Instead of waiting until every element of the IND package is complete, the pilot will allow the FDA to review individual components on a rolling basis during the pre-IND stage.

That could give sponsors an opportunity to address problems with manufacturing data, nonclinical studies, clinical plans, or other elements before the formal IND is submitted. In practical terms, the approach is intended to identify and address potential roadblocks earlier in development.

The pilot also encourages sponsors to coordinate other activities that can delay a study, including Institutional Review Board review and clinical trial site activation. Those efforts cannot override the FDA’s safety review, but more advance planning could help a study begin sooner after the agency allows it to proceed.

The FDA says the pilot could inform future policies for modernizing early clinical development. One possible longer-term outcome would be a formal accreditation model for research institutions that demonstrate strong scientific and regulatory judgment.

Such a system could create a trusted network of institutions capable of helping sponsors prepare high-quality IND applications, particularly in areas where development is scientifically complex or where sponsors do not have extensive in-house experience.

Photo: Acting FDA Commissioner Kyle Diamantas