FDA Opens Expedited IND Pilot With Rolling Pre-IND Review to Cut First-in-Human Timelines
核心洞察
The FDA has finalized the design of its Expedited IND Pilot under Operation Trailblazer (搜索) and opened applications from sponsors and qualified research institutions through October 30.
The pilot replaces single-package IND submission with rolling pre-IND review, with the FDA selecting an initial cohort of 8 to 10 Sponsor-QRI pairs.
US first-in-human development can take up to two years versus a few months in China and Australia, and the pilot aims to cut six to 12 months.
The FDA has published the final design of its Expedited Investigational New Drug (IND) Pilot and opened applications for participation, advancing a program intended to shorten the time from drug discovery to first-in-human phase 1 trials in the United States. The initiative is a key component of Operation Trailblazer (搜索), the Department of Health and Human Services effort launched in June to reduce red tape and reverse the offshoring of early-phase clinical research.
The pilot responds to a competitive gap: first-in-human development in the US can take up to two years, while comparable early-stage trials move more quickly in markets such as Australia and China. China has now overtaken the US in the number of registered clinical trials. The FDA aims to slash US timelines by six to 12 months.
Rolling Review Replaces Single-Package Submission
The core mechanism replaces the conventional model — in which a complete IND application is submitted before any FDA review begins — with a rolling pre-IND review conducted in partnership with qualified research institutions (QRIs). Drug sponsors and prospective QRIs apply as a pair, and the FDA will select an initial cohort of 8 to 10 Sponsor-QRI pairs.
Selected QRIs are expected to bring specialized scientific expertise to IND preparation, enabling the agency to review and accept individual components as they are completed rather than waiting for a full package. The pilot also encourages parallel coordination of activities such as Institutional Review Board review and clinical trial site activation during IND development, with the aim of reducing delays between IND preparation and the start of first-in-human studies.
The FDA retains full regulatory authority throughout, including the ability to impose clinical holds and determine whether an investigation may proceed. By enabling real-time identification and resolution of IND deficiencies, the pilot is intended to reduce the risk of a clinical hold during the standard 30-day IND review period.
The IND process involves review of phase 1 trial protocols used to determine dosing, absorption, and initial safety in humans.
Timelines and Selection
The FDA will accept applications to participate until October 30 and intends to notify successful applicants by December 18, with the initial cohort of 8 to 10 Sponsor-QRI pairs selected by that date, although the timeline may vary depending on application volume.
"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.
"Under the Trump Administration, boosting domestic innovation and ensuring American patients have first access to groundbreaking treatments is a top priority," he added. "The FDA will continue implementing Operation Trailblazer (搜索) to modernise regulatory processes to keep our country ahead of global competitors without compromising patient safety."
Toward an Accreditation Model
According to the agency, the pilot will generate evidence to inform potential future approaches to modernising FDA support for early-stage clinical development. The FDA has suggested this could lead to a formal accreditation model for QRIs and create a network of institutions to support drug development and accelerate the time to first-in-human trials — a more durable structural change that would move US IND support toward a recognized network of institutions rather than ad hoc sponsor-led preparation.
Mwango Kashoki, head of regulatory science at contract research organisation Parexel, said the pilot could lead to a "meaningful shift" in the use of phase-appropriate data and greater flexibility in how evidence is structured.
"At its core, the programme will enable sponsors to generate more decision-relevant data earlier, improving clarity at the point of first-in-human evaluation," she said. "Over time, this approach has the potential to streamline development pathways while maintaining a strong scientific foundation, ultimately supporting faster, more efficient translation from drug discovery to clinical testing."
A Broader Regulatory Modernization Push
The Expedited IND Pilot is one of several regulatory modernization efforts active simultaneously. The FDA launched its PreCheck Pilot in February to accelerate domestic manufacturing facility development, selecting seven initial participants in June. Separately, China's Center for Drug Evaluation (搜索) introduced its PIONEER Program in August for cell and gene therapy development, underscoring that regulatory acceleration has become a competitive priority across multiple jurisdictions.