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  1. FDA Actions to Accelerate and Modernize Early and Late-Stage Clinical Development

FDA Expedited Investigational New Drug (IND) Pilot Program

Accelerating Early-Stage Clinical Research in the U.S.

Program Overview | Rolling Submission Process & Structure | Application Instructions

The FDA Expedited IND Pilot Program is an initiative under HHS Operation TrialBlazer designed to evaluate new approaches for accelerating first-in-human (FIH) clinical trials in the United States while maintaining FDA's rigorous standards for participant safety and scientific oversight. 

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The pilot seeks to reduce the time from initiation of IND-enabling activities to FIH trial initiation by testing a collaborative model in which sponsors work with prospective qualified research institutions (QRIs) to support more efficient drug development. Under the pilot, third parties (which could include academic medical centers, health networks, contract research organizations, regulatory advisors, or other research organizations), collectively called “QRIs”, would partner with sponsors to provide expert recommendations on pharmacology and toxicology, clinical, and chemistry, manufacturing, and controls (CMC) components of first-in-human IND submissions. These QRIs should function as scientific experts providing risk-proportionate judgement to drive innovative, science-based, phase-appropriate regulatory standards.  

Because QRIs bring specialized and substantial scientific expertise to the preparation process, the FDA will accept and review individual components of an IND submission on a rolling basis as they are completed, rather than waiting for a complete package. This rolling review model has the potential to fundamentally change the timeline of pre-IND drug development. Issues can be identified and resolved in real time, the risk of a clinical hold at the end of the process may decrease, and the path from scientific discovery to FIH trial will potentially become faster, more predictable, and more collaborative. Sponsors retain ownership of and responsibility for their IND component submissions throughout the process.

The pilot also seeks to encourage coordination of activities that may occur alongside IND development and FDA review, such as Institutional Review Board (IRB) review and clinical trial site activation, where appropriate. By supporting earlier planning and more coordinated development activities, the pilot aims to reduce unnecessary delays between the point at which a sponsor begins preparing for an IND submission and the start of first-in-human studies.

Participation in the pilot is voluntary. FDA retains full regulatory authority throughout the program, including responsibility for determining whether a clinical investigation may proceed, whether a clinical hold should be imposed, and all other regulatory oversight activities. Participation in the pilot does not modify FDA's statutory or regulatory standards for IND review, safe-to-proceed determinations, or clinical trial conduct.

The Expedited IND Pilot Program is intended to evaluate whether leveraging QRIs and a new rolling review process can improve the efficiency of early clinical development while maintaining FDA’s rigorous standards for participant safety and scientific oversight.  

Through this pilot, FDA seeks to assess whether the following outcomes can be achieved:

  • Improved quality and submission of phase-appropriate data for FIH 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.
  • Greater coordination of clinical trial startup activities, including IRB review and clinical trial site readiness, where appropriate.  

In addition to evaluating whether this collaborative approach improves submission quality, regulatory efficiency, and early clinical development timelines, the pilot will generate evidence to inform potential future approaches to modernizing FDA’s support of early-stage clinical development. For example, this pilot may inform future policy initiatives that could certify or accredit QRIs demonstrating strong scientific and regulatory judgment with the goal of building a sustainable, high-quality network of institutions to support drug development and accelerate the time to FIH trials.

The United States has long set the global standard for pharmaceutical innovation and regulatory rigor. But in recent years, a growing share of early-stage clinical research has moved overseas, threatening America’s position as the global leader in biomedical innovation. It is critical to protect clinical research in the U.S. because it ensures that American patients will continue to have earlier access to new therapies and remain protected by the world's most advanced regulatory oversight.

Early clinical development plays a critical role in bringing new therapies to patients. Sponsors that reach first-in-human milestones efficiently are often better positioned to secure additional investment and establish partnerships critical to the development of safe and effective drugs, as well as advance promising products through later stages of development.

Strengthening the foundation for successful Investigational New Drug, or IND, submissions begins at the pre-IND stage. As drug development has grown increasingly complex and diverse across both drugs and biologics, the volume and sophistication of scientific questions sponsors must resolve is testing the limits of the current pre-IND framework, yet there is a meaningful opportunity to evolve. The current framework asks a single nonbinding pre-IND advisory meeting to perform an enormous amount of work, addressing nonclinical, clinical, manufacturing, trial design, and patient safety considerations all at once. For complex development questions, that concentration of activity leaves little room for the iterative, structured collaboration sponsors need to build toward a strong submission. Expanding opportunities for that kind of engagement would better position sponsors to bring safe and effective therapies to patients more efficiently.

Sponsors must then complete trial site activation activities, such as institutional review board review, site contracting, and patient enrollment. Currently, these activities are often completed sequentially when they could be pursued in parallel, which could further compress trial activation timelines.

As stated in HHS Operation TrialBlazer, FDA is committed to proactively address clinical development challenges through dedicated initiatives to accelerate and modernize clinical research. First, FDA is focusing on refining IND expectations to reflect what is scientifically appropriate for first-in-human Phase 1 trials, prioritizing patient safety while assessing risk to reduce over-submission. This is not a one-time fix, but an ongoing commitment to ensure phase-appropriate expectations keep pace as modalities and scientific understanding evolve. Second, FDA is exploring new ways to unlock the power of American innovation through the Expedited IND Pilot.

Lessons learned from the pilot may help inform future approaches to supporting efficient first-in-human drug development in the United States.

Participant Characteristics & Conflict of Interest

Sponsors may submit a request to participate for a single IND submission. FDA will prioritize submissions with the following characteristics:

  • The product candidate is novel and will fall under the review jurisdiction of CDER Office of New Drugs (OND), CBER Office of Therapeutic Products (OTP), or Oncology Center of Excellence (OCE).
  • The IND is classified as a Commercial IND, i.e., the sponsor intends to commercialize the product by eventually submitting a marketing application.
  • The IND program targets a Phase 1 First-In-Human IND submission, and the investigational product does not have existing clinical experience.
  • The intended IND submission is for a FIH Phase 1 clinical trial that will be run in the United States
  • The sponsor has sufficient preliminary nonclinical data at the time of application to the pilot program to support the FDA in evaluating the product’s proposed development timeline and IND submission timeframe.  
  • The pilot will address the investigational product as defined in the application. Programs incorporating novel technologies or platforms are not excluded, but pilot support will be limited to the technology as incorporated into this specific IND, not to the platform or technology broadly. 

Prospective QRIs may submit a request to participate in the pilot in support of a single IND submission in partnership with a sponsor. FDA will prioritize QRIs with the following characteristics:

  • The prospective QRI is a legal entity organized or incorporated in the United States, with a principal place of business in the United States, with core advisory personnel located in the United States.  
  • The prospective QRI commits to providing IND development or advisory support across nonclinical, CMC, and clinical disciplines for the IND program identified in the sponsor’s application.  
  • The prospective QRI commits to supporting the acceleration of Phase 1 FIH clinical trial initiation in the United States through owned infrastructure or formally executed and documented partnership arrangements. 

FDA will maintain full regulatory authority and conduct thorough, independent review of all IND submissions during the Expedited IND Pilot. Conflict of interest between sponsors and QRIs is the responsibility of the participating organizations to identify and manage. FDA expects QRIs to implement written COI procedures that should be available to FDA upon request.  

FDA will evaluate pilot participants based on the quality and objectivity of IND submissions and QRI recommendations. Should conflict of interest be found to have compromised a QRI's ability to provide objective recommendations, the QRI-sponsor pair will be removed from the pilot, and FDA may consider this experience when evaluating the QRI model .  

Examples of topics that may be included in the written conflict of interest procedures include:

  • Identification and disclosure of financial, employment, collaborative, and personal relationships between QRI personnel and the sponsor or the sponsor’s competitors;  
  • A process for determining whether identified real or potential conflicts are problematic or manageable;  
  • For any potential conflicts of interest that can be managed, procedures for doing so.

QRI advisory team & IRB Conflict of Interest: In the case of a prospective QRI that also owns or operates an IRB that will review the clinical protocol associated with the pilot IND, the QRI must have written procedures ensuring that IRB members do not hold dual membership or roles within the QRI's pilot advisory process. This separation must be maintained throughout the pilot period. An IRB may, at its discretion, invite individuals with competence in special areas, including subject matter experts from the QRI, to assist in the review of complex issues which require expertise beyond or in addition to that available on the IRB. These individuals may not vote with the IRB (as per 21 CFR 56.107(f)).

Selection Considerations

FDA will evaluate sponsor development programs and QRI qualifications against the following considerations for participation in the Expedited IND Pilot Program. Applications will be evaluated as a single package across both parties in tandem (the sponsor and QRI).

Because the pilot aims to include a variety of QRI types and IND submissions spanning novel mechanism programs and more established approaches across diverse therapeutic areas, applications will be evaluated relative to one another. FDA will seek to ensure the selected cohort reflects breadth across therapeutic areas, product modalities, sponsor sizes, and QRI types. Because of this, no single factor is determinative and will be evaluated holistically across the applications received. Sponsors should note that non-selection for the pilot will not disadvantage sponsors in any way, disqualify sponsors from utilizing typical pre-IND meetings or other FDA interactions, nor does it imply lower-quality drug development research. 

  • IND Complexity & Pilot Fit: The pilot is designed to test the rolling submission model across a range of modalities and program types. Applications will be assessed on whether the IND's complexity and modality are well-suited to the rolling submission format and whether the proposed QRI partnership adds meaningful value to the development program within the pilot timeline.  
  • IND Development Stage: FDA will assess whether the IND program is at a stage where QRI engagement can be substantively productive within a reasonable timeframe. The CMC and nonclinical development program should be sufficiently advanced so that an IND submission is credibly achievable within a reasonable timeframe, while early enough that the QRI can provide meaningful input, whether on study design, data package strategy, or submission readiness, rather than serving primarily as an advisor on a near-final package. Applicants will be asked to describe the status of their proposed nonclinical program, including CMC, and provide a high-level clinical protocol synopsis in the pilot application.
  • Public Health Impact & Unmet Medical Need: FDA will consider the severity and burden of the target condition and the availability of existing treatments.  
  • Sponsor Profile: The pilot is designed to engage both small and emerging biotechnology companies and larger sponsors with established IND experience, in order to test the different forms of value a QRI may provide, whether as an active development partner throughout the IND preparation process, or as a substantive preliminary reviewer of IND components during rolling review. Sponsors will be asked to describe their IND submission experience and explain which model of QRI engagement they anticipate, and why.  
  • Portfolio Considerations: Because the pilot is designed to generate learning across the drug development ecosystem, the considerations above will be evaluated holistically and in the context of the full applicant pool. Applications representing underrepresented therapeutic areas, rare diseases, pediatric indications, or novel modalities may contribute value to cohort diversity and will be considered accordingly.  

Prospective QRIs will be evaluated on their documented experience across the following core disciplines, which are necessary to effectively support the sponsor throughout the IND development and submission process. Multiple areas of expertise may be satisfied by a single person.

  • Nonclinical Leadership & Expertise: Proven capability in designing pharmacology and toxicology data packages, advising on starting dose selection, and assessing whether a nonclinical package will be fit-for-purpose for a Phase 1 IND submission. QRIs should be able to identify gaps and/or potential safety concerns early in the nonclinical program and provide actionable guidance to sponsors on how to address them within a defined timeline.
  • Chemistry, Manufacturing, and Controls (CMC) Leadership & Expertise: Relevant background in guiding process and analytical development and characterization strategies, identifying critical CMC gaps, and developing the CMC module of an IND for safety and regulatory adequacy. QRIs should be able to assess whether a sponsor's manufacturing and controls approach is appropriate for the stage of development and flag issues that complicate FDA review.
  • Clinical Leadership & Expertise: Board-certified physician expertise (MD/DO) with direct experience in Phase 1 clinical trial design, including the development of safety monitoring plans and patient eligibility criteria. Experience serving on or coordinating with Institutional Review Boards (IRBs) could provide value to the sponsor.
  • Clinical Pharmacology: Where applicable to the IND program, QRIs should have documented clinical pharmacology expertise, such as a PharmD or dedicated clinical pharmacologist  to guide or evaluate the pharmacokinetic and pharmacodynamic components of the IND submission, including any modeling approaches intended to contribute to dose selection.  
  • Regulatory Affairs Leadership & Expertise: Personnel with direct experience managing or reviewing requirements for regulatory submissions, and coordinating integrated nonclinical, clinical, and CMC development timelines.  
  • Program-Specific Expertise: Depending on the specific nature of the IND program, the QRI may be expected to utilize additional, discipline-specific experience for novel therapies, rare diseases, or other modalities.
  • Clinical Phase 1 Trial Support: QRIs should have either self-owned clinical trial infrastructure or documented partnership arrangements with IRB-approved clinical trial sites sufficient to support site activation activities, including IRB review, contracting, and site readiness, in parallel with IND review.  

FDA recognizes that many prospective qualified research institutions may not have all the expertise described above within their own organization and encourages institutions to apply alongside a sponsor even where gaps exist. Prospective QRIs may supplement their in-house capabilities by contracting with external subject matter experts or organizations to fill specific expertise gaps necessary for the indication and therapeutic area of the investigational new drug application. The prospective QRI will remain primarily responsible for determining readiness of the sponsor’s submission and coordinating additional support as required.

Sponsors may continue to engage other external experts, such as Contract Research Organizations or Contract Development and Manufacturing Organizations, to support their development program during the pilot, but may apply with only one designated QRI.  

Submission Details & Next Steps

FDA will use responses to the forms found on the Application Instructions tab to select Expedited IND Pilot Program participants.  

Timeline of Pilot Program Selection:  

  1. September 15: FDA launches Expedited IND Pilot Program and opens window for sponsors to submit applications.
  2. *October 30: Deadline for sponsors to submit Expedited IND Pilot Program applications.
  3. *December 18: FDA selects cohort of Expedited IND Pilot Program participants and notifies all applicants of selections.  

Sponsors who are not selected in the pilot program cohort are encouraged to utilize existing regulatory interactions, such as INTERACT and/or Pre-IND meetings, to ask questions of the FDA as they prepare for IND submission.

*Note: Timelines may vary depending on the number of submissions received. 

Frequently Asked Questions (FAQs)

Please refer to the webinar that was released on August 6, 2026, for additional information regarding the pilot.

If you have additional questions regarding the Expedited IND Pilot, please send to ExpeditedINDPilot@fda.hhs.gov, and the FDA will try to respond as soon as possible. 

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