An Express Lane To Clinical Testing? FDA's Expedited IND Pilot Program

By Luke Nguyen

On average, the length of time between a sponsor’s pre-IND meeting and final IND submission is more than a year. Accounting for the full spectrum of IRB and contractual requirements, this timeframe frequently balloons to two full years from the pre-IND meeting to the initiation of the Phase 1 trial.¹ This timeframe well exceeds the processes in other countries, such as China, which requires IND applicants to initiate the trial within 12 weeks of the applicable IND submission.² Other countries’ reduced timeframes have led more and more sponsors to initiate Phase 1 trials abroad.

This shift could have consequences for patients in the United States. For example, in a recent webinar, FDA noted that such a shift could lead to less representative trial data; delays in patient access; and a reduction in the ability to respond to future emergencies (e.g., pandemics). 

To reduce delays in early-stage drug development and entice early-stage trials to return to the United States, the Department of Health and Human Services (HHS) launched “Operation TrialBlazer.” A key part of Operation TrialBlazer is FDA’s Expedited IND Pilot Program (Pilot Program), which will test a new method of IND review in a selected cohort of sponsors. While far from a guaranteed future pathway, the Pilot Program is a significant step in modernizing FDA’s IND pathway.

The (Potential) New Approach

The Pilot Program seeks to pair sponsors with Qualified Research Institutions (QRIs) early in the preclinical phase to streamline and improve IND submissions. Sponsors will be able to meet with QRIs on an ongoing basis, increasing the opportunity for real-time feedback and collaboration to expedite preparation of the full IND package. This should also limit the need for extensive, often time-consuming modifications to the pre-IND program that can follow a pre-IND meeting or FDA’s review of the full IND package. In addition to the introduction of QRIs as resources in the pre-IND phase, FDA believes that the Pilot Program will improve efficiency by using a rolling IND submission process. In particular, the rolling IND submission process will allow the sponsor to submit components of the IND to FDA with questions and requests for feedback, and request meetings with FDA following development of individual components. As such, sponsors can receive feedback on the nonclinical; clinical; and chemistry, manufacturing, and controls (CMC) components and make any necessary updates before submitting the final IND package for review.

This is a major shift from the current IND process, in which sponsors have only one pre-IND meeting opportunity with FDA, which typically occurs once the IND package is nearly complete, and do not have QRIs to lean on during the pre-IND stage. The increased feedback from QRIs and FDA throughout the preclinical stage should allow FDA to expedite the review of the full IND package and, hopefully, reduce the 30-day window between submission and issuance of a “safe to proceed” notification or clinical hold letter.³

Separately, sponsors will be expected to receive IRB review prior to IND clearance. According to FDA, the goal is for the IRB review to occur concurrently with FDA’s review of the final IND package. For instance, once there is a draft clinical protocol, the draft protocol will be submitted to the IRB, rather than waiting for FDA to conclude its review. Currently, most IRB reviews occur after IND clearance, which can result in additional delays before initiation of the proposed clinical trial.

Understanding Qualified Research Institutions (QRIs)

QRIs are intended to act as scientific partners with the sponsor in developing an IND program. Primarily, the QRIs will be third parties, such as academic medical centers, contract research organizations, or regulatory advisors that provide expertise across the core components of the IND program. Specifically, FDA will expect QRIs to demonstrate experience in the nonclinical, clinical, and CMC components of the IND, as well as offer sponsors Phase 1 clinical trial support, such as site activation activities.⁴

In the Pilot Program, FDA plans to allow prospective QRIs to apply for participation in partnership with a sponsor application. However, should the Pilot Program be permanently adopted by FDA, the final qualification criteria for QRIs will likely be informed by a review of the Pilot Program IND applications. For example, if a Pilot program application has significant issues that hinder the efficiency of FDA’s review of an IND and/or result in a full clinical hold, this could indicate that the sponsor did not receive adequate advice from its QRI.⁵

Of course, regulatory decision-making concerning the IND remains solely within the jurisdiction of FDA, and QRIs will act in an advisory capacity to the sponsor.

Key Objectives Of The Pilot Program

The overarching goal of the Pilot Program is to assess the utility of QRIs and the rolling IND submission process as an effective method to improve the efficiency of early-stage drug development. In particular, FDA plans to assess the following outcomes in determining the success of the Pilot Program:

• Improved quality of IND submissions;
• Reduction in the number of Phase 1 clinical holds and information requests;
• Earlier identification of scientific and regulatory issues through increased QRI and FDA engagement; and
• Reduction in time to initiate Phase 1 clinical trials.⁶

FDA plans to develop success metrics to measure these desired outcomes in the near future, incorporating industry feedback received following its Request for Information earlier this year.

What’s Next For The Pilot Program And Future INDs?

Applications for participation in the Pilot Program close on October 30, 2026, with FDA aiming to finalize the Pilot Program participants by December 18, 2026. The Pilot Program prioritizes applications in which the intended IND submission is for a First-in-Human Phase 1 clinical trial run in the United States. In addition, FDA plans to prioritize commercial INDs intended to support new molecular entities. This means that products involving new delivery systems, formulations, dosage forms, or routes of administration, as well as generic products, are not eligible for the Pilot Program.⁷ Similarly, INDs for Phase 2 or Phase 3 trials are not eligible for the Pilot Program.

Of course, whether FDA permanently adopts an expedited IND program — and the specific confines of such program — will largely depend on the information learned from the first cohort of Pilot Program participants. Even if FDA opts against permanently adopting an expedited IND program, improved efficiency in the drug development process is front-of-mind and signals that at least some of the much-needed changes to the U.S. drug development process may be on the horizon. Interestingly, in FDA’s webinar on the Pilot Program, it was noted that FDA has been in contact with agencies in other countries to further understand early-stage pathways that may help expedite the IND process in the U.S. without sacrificing safety standards.⁸

As the Pilot Program commences, non-participants should continue to operate under the current IND process, with a keen eye toward FDA’s updates on the success of the Pilot Program, as well as announcements on potential alternative methods to accelerate early-stage clinical trials. In addition, sponsors looking to independently expedite the early development process may consider implementing measures similar to those employed in the Pilot Program on their own (e.g., engaging with QRI-like advisors from the preclinical phase onward) to help avoid incomplete or deficient submissions resulting in information requests and/or a clinical hold.

About The Author:

Luke Nguyen is an associate in Haynes Boone’s Dallas office, where he focuses on FDA regulatory compliance for companies developing, manufacturing or marketing FDA regulated products. He can be reached at Luke.Nguyen@haynesboone.com.

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