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

The U.S. Food and Drug Administration has launched the final version of its Expedited Investigational New Drug (IND) Pilot, opening applications for a program designed to shorten the pathway from drug discovery to first-in-human clinical trials conducted in the United States. Announced September 15, 2026, the initiative is part of the Trump Administration's broader push to accelerate clinical research, strengthen domestic drug development and preserve U.S. leadership in biomedical innovation.

Applications for the initial cohort are open through October 30, 2026, with the FDA expecting to select and notify approximately 8 to 10 sponsor-QRI pairs by December 18, 2026. Participation is voluntary, and companies not selected retain access to existing mechanisms such as INTERACT and pre-IND meetings.

A Rolling Review Model in the Pre-IND Phase

The pilot's central feature is a rolling review model during the pre-IND phase. Rather than waiting until every component of an IND application is complete, the FDA will review individual sections as they become ready. The agency says this is intended to allow regulatory or scientific problems to be identified earlier in development rather than during the formal IND review period.

By resolving questions in real time, the FDA hopes to improve submission quality, reduce the risk of clinical holds during the 30-day IND review period, and make the pathway to first-in-human testing faster, more predictable and more collaborative. The agency emphasized that the program does not change FDA safety standards or regulatory authority: the FDA retains full responsibility for determining whether a clinical investigation may proceed, whether a clinical hold should be imposed, and all other regulatory oversight.

Under the model, drug sponsors partner with Qualified Research Institutions (QRIs) that provide scientific expertise during preparation of the IND application. These organizations may include academic medical centers, health systems, contract research organizations, regulatory advisers and other research institutions capable of contributing expertise in pharmacology and toxicology, clinical development, and chemistry, manufacturing and controls. An FDA spokesperson said ahead of the announcement that academic research centers as well as contract research organizations could fit the QRI description, and that the pilot is open to all eligible sponsors, including smaller companies and startups.

"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, JD.

Karim Mikhail, BPharm, MS, Director of the FDA's Center for Biologics Evaluation and Research (搜索)搜索该词条 (CBER), said: "The pilot hopes to utilize the American innovation ecosystem to accelerate the time to first-in-human clinical trials. 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."

Eligibility and Selection Priorities

The FDA will prioritize novel product candidates expected to fall under the review jurisdiction of the Center for Drug Evaluation and Research (搜索)搜索该词条 Office of New Drugs, the Center for Biologics Evaluation and Research (搜索)搜索该词条 Office of Therapeutic Products, or the FDA Oncology Center of Excellence (搜索)搜索该词条. Eligible programs must target a Phase 1 first-in-human IND submission, involve an investigational product without previous clinical experience, and plan to conduct the first-in-human study in the United States.

Selection will consider the complexity of the IND, stage of development, public health impact and unmet medical need. The agency plans to build a diverse cohort spanning therapeutic areas, product modalities, sponsor sizes and QRI types, and notes that programs involving rare diseases, pediatric indications, novel modalities and underrepresented therapeutic areas may contribute to that diversity. These criteria could make the pilot relevant to developers of new anticancer drugs, biologics, cell and gene therapies and other experimental oncology platforms entering first-in-human development.

Addressing Offshoring of Early-Stage Research

The initiative comes amid concern within the U.S. government that an increasing proportion of early-stage clinical research is moving outside the country. According to the FDA, first-in-human clinical trials may currently take up to two years to complete in the United States, while similar studies can move considerably faster in countries including China and Australia. In Australia, trials can begin less than 70 days after submission of a study protocol, aided by financial incentives and streamlined reviews, and China-run studies rose from just under 1,000 annually in 2010 to more than 5,000 in 2024, surpassing a stagnant rate of about 3,500 per year in the U.S., according to a National Bureau of Economic Research paper cited by BioPharma Dive. More than 100 licensing deals have been signed between a U.S. or European biopharmaceutical company and a Chinese-based drug developer since the start of 2025, per BioPharma Dive data.

"When early stage clinical development shifts abroad, America risks losing investment, intellectual property, top scientific talent, and most importantly, early access to life-saving therapies for American patients," said Michael Davis, MD, PhD, Director of the FDA's Center for Drug Evaluation and Research (搜索)搜索该词条, speaking to reporters.

Davis framed the pilot as "a direct response to a challenge we hear consistently from drug sponsors: the time and uncertainty involved in moving from scientific discovery to first-in-human trials." He added that the FDA greatly appreciated public feedback on the proposed pilot and incorporated it into the final design, and that the agency "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."

Beyond the Regulatory Review

The pilot also targets delays occurring outside the formal review process. Sponsors and participating institutions will be encouraged to coordinate activities such as Institutional Review Board review and clinical trial site activation alongside IND development and FDA review where appropriate. Normally, some of these activities occur sequentially, adding time between IND preparation and enrollment of the first patient. Mikhail said the program is meant to loosen bottlenecks by ensuring trial site preparation and ethics reviews occur "in parallel" rather than "in sequence."

The experience gained could shape broader reforms. The FDA has indicated that findings could inform future policies, potentially including a formal accreditation or certification model recognizing QRIs that demonstrate strong scientific and regulatory judgment, aimed at creating a sustainable, high-quality network of institutions to support drug development.

Industry Reaction

In August, the Biotechnology Innovation Organization (搜索)搜索该词条 (BIO), a lobbying group representing U.S. drugmakers, agreed that a rolling review process would "likely result in significant time savings." However, BIO was skeptical that any single research institution could meet the requirements the FDA set forth, and was concerned that these evaluations might add "an additional layer of review" and slow progress.

The launch marks the transition of the Expedited IND initiative from a proposed regulatory experiment into an active FDA program. Its first cohort will provide an early test of whether closer collaboration between regulators, sponsors and research institutions can meaningfully shorten first-in-human drug development while maintaining existing safety and scientific standards.