About the pilot program
The FDA Expedited IND Pilot Program is an initiative under HHS Operation TrialBlazer designed to evaluate new approaches (namely leveraging QRIs and a new rolling review process) for accelerating first-in-human (FIH) clinical trials in the United States while maintaining FDA’s rigorous standards for participant safety and scientific oversight.
The pilot seeks to:
1. 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 any of the following 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.
- academic medical centers
- health networks
- contract research organizations
- regulatory advisors, or other research organizations).
- These QRIs should function as scientific experts providing risk-proportionate judgement to drive innovative, science-based, phase-appropriate regulatory standards.
2. 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.
What are the desired outcomes of the pilot?
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.
Is participation in the program voluntary?
Yes it is.
- 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.
Under what considerations will the FDA evaluate sponsor development programs and QRI qualifications for participation in the program?
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).
i) Sponsor IND considerations
- 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.
ii) Prospective QRI selections
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.
Which single IND submissions will be prioritised for the program?
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.
- FDA will prioritize new molecular entities without established clinical experience.
- Therefore, FDA recommends sponsors do not submit a request to participate in this pilot for an investigational product that leverages active ingredients with prior clinical experience.
- Products such as those involving new formulations, delivery systems or technologies, drug-device combinations, routes of administration, dosage forms, or development under the 505(b)(2) pathway are not the primary focus of the pilot.
- 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.
Which qualified research institutions (QRIs) will be prioritised for the program?
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.
Source: FDA
Timeline of the pilot:
| Date | Event |
|---|---|
| 22 Jun 2026 | The FDA introduced the proposed Expedited IND Pilot Program under HHS’s Operation TrialBlazer. |
| 15 Sept 2026 | FDA launched the Expedited IND Pilot Program and opened the window for sponsors to submit applications. |
| 30 Oct 2026 | The deadline for drug sponsors and prospective Qualified Research Institutions (QRIs) to submit joint applications. |
| 18 Dec 2026 | The FDA will select cohort of Expedited IND Pilot Program participants and notifies all applicants of selections. |
What types of investigational products are the pilot’s primary focus?