The Expedited IND Pilot Program advances the Department of Health and Human Services’ (HHS) Operation TrailBlazer initiative, which is aimed at implementing several strategies to “foster a clinical research economy that delivers timely, safe, and effective medical products to support a healthy America."

The U.S. Food and Drug Administration (FDA) recently announced the final design of its much-anticipated Expedited Investigational New Drug (IND) Pilot Program. To participate in the program, sponsors and prospective Qualified Research Institutions (QRIs) may submit applications until October 30, 2026, and FDA will select its cohort of Pilot Program participants by December 18, 2026. Timelines may vary depending on the number of submissions FDA receives. The FDA expects to select eight to 10 sponsor-QRI pairs for the initial pilot cohort.

The Expedited IND Pilot Program advances the Department of Health and Human Services’ (HHS) Operation TrailBlazer initiative, which is aimed at implementing several strategies to “foster a clinical research economy that delivers timely, safe, and effective medical products to support a healthy America.” Through the Expedited IND Pilot Program, FDA will facilitate two strategic priorities of Operation TrailBlazer: (1) accelerating the time to first-in-human clinical trials by addressing the regulatory requirements for IND applications, effectively reimagining the pre-IND phase, and (2) accelerating later-stage clinical development and reducing administrative burden by pursuing significant Institutional Review Board (IRB) reform and increasing trial access and participation. 

The Expedited IND Pilot Program has been designed to help the United States hold its position as a global leader in pharmaceutical innovation, biomedical research and regulatory rigor. The current regulatory landscape in the United States oftentimes causes the clinical trial phase to be unnecessarily extensive, therefore inhibiting innovation and causing a delay in bringing new therapies to patients. In the United States, first-in-human Phase 1 clinical trials can take up to two years to complete, largely due to early development and IND application bottlenecks.

In turn, competitors of the United States in biomedical innovation are able to get cutting-edge treatment to patients more quickly. For example, China and Australia have implemented streamlined regulatory pathways to accelerate Phase 1 clinical trials, which has resulted in both countries becoming world leaders in biotechnology and clinical research. For both foreign competitors, early discovery-to-IND cycles are 50 to 70 percent faster than the rest of the world, including the United States. As a result, pharmaceutical and biotech companies, also called “sponsors,” gravitate towards these countries for development and commercialization to save time, money and avoid increased regulatory red tape.

To similarly streamline development in the United States, the Expedited IND Pilot Program will use QRIs, which may include contract research organizations, health networks, medical centers, or regulatory advisors. In the program, QRIs will partner with sponsors to develop expert recommendations on pharmacology and toxicology, clinical, and chemistry, manufacturing and controls (CMC) components of first-in-human IND submissions. These QRIs have specialized and substantial scientific expertise, which allows them to provide risk-proportionate judgment and serve as a review and advisory resource while, at the same time, allowing sponsors to retain full ownership of their IND. FDA will also employ collaborative coordination to IND development and FDA review by including the Institutional Review Board (IRB) review and clinical site activation.

Simultaneously, FDA will establish a new, real-time rolling submission platform to allow it to review QRI recommendations and IND submission components on a rolling basis. To provide timely guidance, FDA will also be able to communicate securely with the sponsor. This method will allow the review of individual IND components prior to the formal IND submission. By not waiting for all IND components to be complete to begin review, the agency will fundamentally change the timeline of pre-IND development.

FDA will continue to uphold its statutory and regulatory standards for IND review to protect and promote public health while assessing risk to reduce over-submission. With the results of the Expedited IND Pilot Program, FDA will assess the achievement of certain outcomes, including:

• Improved quality and submission of phase-appropriate data for first-in-human IND submissions through structured, multidisciplinary scientific input across nonclinical, clinical and CMC disciplines;
• Earlier identification and resolution of scientific and regulatory issues that may delay initiation of Phase 1 clinical trials;
• Reduced instances in which Phase 1 clinical holds or information requests may be necessary through higher-quality submissions and earlier issue identification;
• More efficient progression through nonclinical development to first-in-human study initiation; and
• Greater coordination of clinical trial startup activities, including IRB review and clinical trial site readiness, where appropriate.

Fundamentally, the Expedited IND Pilot Program will inform FDA about potential future methods to modernize its approach to early-stage clinical development, including the creation of policy initiatives. The Expedited IND Pilot Program is voluntary, and FDA retains full regulatory authority throughout the program.

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