FDA Outlines IND Pilot to Speed Early Clinical Trials, Counter Overseas Competition

FDA is continuing to outline its intention to overhaul the early-stage clinical development process through a planned Expedited IND Pilot, arguing that the changes are needed to prevent drug development from shifting overseas and to accelerate first-in-human clinical trials in the U.S.

In an 8/5 FDA Voices post, acting CBER director Karim Mikhail said the agency is seeking to modernize a regulatory process that he described as outdated and increasingly unable to support today's complex drug development landscape, particularly for advanced therapies such as cell and gene therapies.

Mikhail said lengthy U.S. development timelines are placing the country at a competitive disadvantage. According to FDA, Phase 1 clinical trials that can take up to two years to begin in the U.S. are being completed in about nine months in China, prompting concerns that investment, intellectual property and scientific talent could increasingly migrate abroad. He said the initiative aligns with directives from the Trump administration to reduce regulatory barriers and strengthen U.S. leadership in biomedical innovation.

The agency identified several shortcomings in the current IND process, including uncertainty over what data are required for first-in-human studies, a pre-IND meeting structure that often provides only a single opportunity for sponsors to obtain FDA feedback, and delays that occur after IND clearance because of institutional review board approvals, site contracting and trial activation.

To address those issues, FDA plans to clarify what information is appropriate for Phase 1 IND submissions to reduce unnecessary data packages and sponsor uncertainty. This will “reflect what is scientifically appropriate for first-in-human (FIH) Phase 1 trials, prioritizing patient safety while eliminating the ambiguity that drives over-submission,” Mikhail said. “This is not a one-time fix, but an ongoing commitment to ensure phase-appropriate expectations keep pace as modalities and scientific understanding evolve.”

The agency also intends to rely on its Expedited IND Pilot that would enlist Qualified Research Institutions (QRIs) — including academic medical centers, contract research organizations and other scientific organizations — to work closely with sponsors during IND preparation. Under the proposed model, QRIs would provide iterative scientific and regulatory guidance before submission, while FDA would review portions of an IND on a rolling basis rather than waiting for a complete application. The agency said this approach would allow potential scientific or regulatory issues to be identified earlier, reduce the likelihood of clinical holds and shorten the timeline to first-in-human studies without compromising patient safety.

The pilot would also explore whether QRIs could help accelerate activities that occur after IND authorization by initiating institutional review board reviews, site contracting and trial activation in parallel with FDA review, potentially reducing delays before patient enrollment begins.

FDA recently issued a Request for Information seeking public feedback on the pilot's design, including the role of QRIs, the structure of rolling submissions and measures of success across different therapeutic areas. The agency also plans to hold an 8/6 stakeholder Webinar to explain the proposal and answer questions before collecting comments.