FDA launches pilot program to accelerate early-stage drug research in the U.S.

The U.S. Food and Drug Administration (FDA) has formally launched a pilot program to accelerate early-stage clinical trials of new drugs.

According to BioPharma Dive on the 15th (local time), the FDA launched the "Expedited Investigational New Drug (IND) Pilot Program" that day to accelerate the entry of drug candidates into human clinical trials.

The initiative is part of Operation TrialBlazer, first announced by the Department of Health and Human Services (HHS) in June. It aims to reverse the trend of drug companies starting clinical trials overseas, including in China and Australia, where regulations are more favorable.

Drug companies participating in the program will be paired with up to 10 "qualified research organizations" and submit applications to begin clinical trials. The FDA plans to use a "rolling review" process, reviewing application materials as they are received.

The FDA plans to select eight to 10 organization-company pairs for the pilot program, with applications due Oct. 30.

Mike Davis, director of the FDA's Center for Drug Evaluation and Research (CDER), said, "This program directly addresses an issue consistently raised by drug developers: the time and uncertainty involved in moving from scientific discovery to first-in-human studies."

It typically takes up to two years to advance a new drug to the clinical trial stage in the U.S. In Australia, by contrast, trials can begin within 70 days of protocol submission, thanks to financial incentives and streamlined reviews.

As a result, the number of clinical trials conducted overseas has surged. According to a paper by the National Bureau of Economic Research (NBER), China-led studies increased from fewer than 1,000 annually in 2010 to more than 5,000 in 2024. Over the same period, the U.S. remained at about 3,500 per year.

Davis said he was concerned that "when early-stage clinical development moves overseas, the U.S. risks losing investment and intellectual property, top scientific talent and, most importantly, early access to treatments that could save the lives of American patients."

Karim Mihail, director of the Center for Biologics Evaluation and Research (CBER), said rolling review "can help identify and resolve issues early and reduce the risk of clinical holds, making the path to first-in-human studies faster and more predictable."

However, the Biotechnology Innovation Organization (BIO), a lobbying group representing the U.S. biotechnology industry, said in August that rolling review "is likely to result in significant time savings," while also raising concerns that a single research organization may struggle to meet FDA requirements and that the evaluation could become an "additional review step" that slows progress.

An FDA spokesperson said both academic research centers and contract research organizations (CROs) may qualify as qualified research organizations, and that the program is open to all eligible drug developers, including small companies and startups.