FDA Proposes Expedited Investigational New Drug Pilot Program to Drive Early Phase Clinical Research in the United States

On June 22, 2026, the U.S. Department of Health and Human Services (HHS) unveiled Operation TrialBlazer, a department-wide effort to accelerate clinical research and development centered in the United States.¹ As part of that initiative, the U.S. Food and Drug Administration (FDA or the Agency) is taking multi-pronged actions to help facilitate early- and late-stage clinical development, including:

1. proposing a pilot program intended to reduce the start-up time for first-in-human (FIH) clinical trials, as discussed herein;
2. clarifying the chemistry, manufacturing, and controls (CMC) expectations for Investigational New Drug (IND) submissions to help sponsors efficiently generate and submit the phase-appropriate data needed to support phase 1 clinical trials; and
3. issuing a significantly revised draft of FDA’s critical Substantial Evidence Guidance that sets out regulatory expectations for sponsors regarding the type and quantity of data and information necessary to meet the statutory standard for “substantial evidence” of effectiveness in support of a drug or biological product application (see Arnold & Porter’s Advisory, here).²

The HHS Office of Inspector General is also evaluating whether to update the safe harbor regulations under the federal Anti-Kickback Statute or the exceptions to the civil monetary penalty provision prohibiting inducements to beneficiaries for remuneration provided to individuals in connection with their participation in clinical trials.³

HHS estimates that Operation TrialBlazer could cut in half the time it takes to conduct clinical trials in the U.S.⁴ FDA believes that targeting the earliest development stage (i.e., Phase 1) alone could shave 6 to 12 months off a drug program’s overall development timeline.⁵

FDA’s proposed Expedited-IND Pilot program is aimed at reversing a trend of clinical research and development moving abroad, namely to China. China surpassed the U.S. for the global share of phase 1 clinical trials in 2021 and has continued to extend its lead in the number of early-stage trials globally.⁶ Operation TrialBlazer recognizes that China is gaining this competitive advantage since streamlining its regulatory pathways and strengthening its clinical trial infrastructure, which attract sponsors and investment for conducting clinical trial research.⁷

Various ideas have spread throughout the government and industry on how to reverse the trend.⁸ U.S. congressmembers have called for legislation that, if enacted, would prohibit FDA from accepting, reviewing, or considering certain clinical data generated in China in support of an IND, noting concerns with patient safety standards, human rights, and independence from government influence.⁹ FDA has asked Congress to create an expedited IND pathway in its fiscal year 2027 budget request.¹⁰ There have been calls for additional reforms to Institutional Review Boards (IRBs),¹¹ to the way that FDA conducts clinical trial inspections in China,¹² and to strengthen human subject protections for clinical trials conducted in China.¹³ Prescription Drug User Fee Act (PDUFA) VIII has included discussions of user fee incentives for domestic drug development—as well as potentially higher fees for applications that do not include domestic clinical trial data.¹⁴ FDA’s new Expedited-IND Pilot program proposes to pull more trials to the U.S. by expediting IND clearance for FIH clinical trials.

A Proposed Path to Expedite the First-in-Human Milestone

On June 24, 2026, FDA opened a request for information (RFI) to solicit stakeholder input on the proposed Expedited-IND Pilot program.¹⁵ The Expedited-IND Pilot program would establish a network of “Qualified Research Institutions” (QRIs) that would assess information required to be included in an initial IND submission (i.e., pharmacology and toxicology, clinical, and CMC information)¹⁶ and make recommendations—potentially as part of a rolling review of IND materials.

While there will undoubtedly be legal questions as to the basis for FDA’s reliance on QRIs, FDA is clear that it would retain full regulatory oversight of the IND submission, including the authority to impose a clinical hold, disqualify an investigator or IRB, conduct clinical trial inspections, and enforce safety reporting requirements.¹⁷ QRIs would only act as a “review and advisory resource” to sponsors (sponsors would remain responsible for their IND submissions).¹⁸ But QRIs would be expected to expedite overall review timelines by improving the quality of IND submissions, reducing the likelihood that FDA imposes a clinical hold, reducing the time for FDA to review INDs, bridging between IRBs and FDA, and potentially making recommendations to FDA that would reduce the regulatory burden associated with IND review.¹⁹ The RFI seeks input regarding the types of tasks QRIs could take on and how these processes might be structured.

As currently proposed in the RFI, QRIs would be responsible for:

• advising and providing written recommendations to sponsors on the nonclinical (pharmacology and toxicology), clinical, and CMC components of sponsors’ IND submissions;
• conducting conflict of interest screening and establishing a formal engagement agreement with the sponsor;
• holding regular meetings with the sponsor and appropriate subject matter experts to discuss IND development progress;
• maintaining records of discussions, recommendations, and interactions with sponsors;
• sharing their recommendations with FDA through the rolling submission platform (discussed below); and
• participating in pilot evaluation activities and supporting parallel activities, such as IRB review and clinical trial site activation.²⁰

To be qualified, a potential QRI would need to demonstrate capabilities, infrastructure, and leadership expertise across nonclinical, clinical, and CMC disciplines, regulatory affairs, and clinical trials relevant to FIH IND submissions and Phase 1 studies.²¹ FDA indicates that QRIs may need to obtain a formal certification from the Agency after the pilot concludes.²²

As part of the RFI, FDA also seeks input regarding a proposed rolling submission platform through which FDA could review QRI recommendations to the completed components of a sponsor’s IND submission before the final IND submission.²³ FDA likens the rolling review process to the rolling review of a New Drug Application or Biologics License Application under existing expedited review programs.²⁴ FDA believes that rolling review would afford the Agency an earlier opportunity to resolve potential deficiencies that would result in a clinical hold or information request, and to sooner issue the sponsor a “safe to proceed” letter authorizing the FIH study to commence.²⁵ Barring a “safe to proceed letter” or clinical hold, a sponsor must wait 30 days from the date that FDA receives an IND to begin a clinical study.²⁶

FDA believes that accelerating the time to reach FIH milestones would also help biopharmaceutical companies secure key partnerships, attract greater investment in biomedical research in the U.S., and bring new therapies and cures to Americans more quickly.²⁷

The RFI Provides Opportunities to Shape the Expedited-IND Pilot Program

FDA seeks extensive stakeholder input on the contours of the Expedited-IND Pilot program and the qualifications and responsibilities of QRIs.²⁸ We suggest that stakeholders consider engaging in the RFI process to offer feedback on the details of the pilot program—some potential areas are highlighted below—before FDA readies the program for a premiere. If finalized, the program’s frameworks may necessitate a shift in thinking in conventional clinical research agreements, including sponsor relationships with IRBs, academic medical centers, and central labs, and require new approaches to delegation of authority documents and clinical research organization arrangements. For example, if adopted, the proposals could lead to a race for centers to become qualified QRIs and for sponsors and other parties to contract with those centers first, raising important questions about capacity, quality, and liability.

Interested parties can submit comments on the proposed Expedited-IND Pilot program through July 22, 2026,²⁹ including comments regarding:

• Scope and scale – e.g., how many QRIs and therapeutic areas/modalities should be included in the pilot program? How long should the pilot program last, or what volume of participation is appropriate before the pilot program is evaluated?
• QRI qualifications and capabilities – e.g., what changes, if any, should be made to the recommended capabilities, infrastructure, and/or leadership expertise for QRIs? Should QRIs be required to have a self-owned and operated IRB and/or clinical trial site? If a QRI also serves as an IRB for a sponsor, how can potential conflicts of interest be prevented?
• Drug eligibility – e.g., which types of products and/or specific diseases or conditions should be considered for the pilot program? How should they be prioritized for participation?
• Pre-IND and IND review process – e.g., what should be the output of QRI advice and review? What information from this review should be submitted to FDA?
• Oversight and accountability – e.g., how should FDA resolve situations in which it disagrees with QRI recommendations?
• Risks to patients – e.g., does the pilot program inadvertently compromise the safety of trial participants, the scientific rigor of the trial, or ethical standards of the trial? How can FDA mitigate these risks?

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If you have any questions, would like more information, or would like to discuss submitting a comment to FDA’s RFI (by July 22, 2026), FDA’s 2026 Draft Substantial Evidence Guidance (by September 22, 2026), or the Office of Inspector General’s RFI (by August 24, 2026), please reach out to one of the authors of this Advisory or your existing Arnold & Porter contacts.