July 21, 2026
Re: Docket No. FDA-2026-N-4699 for “Expedited Investigational New Drug Pilot Program; Request for Information.”
The National Center for Health Research (NCHR) appreciates the opportunity to comment on the proposed Expedited Investigational New Drug (IND) Pilot Program. NCHR is a nonprofit, nonpartisan organization that analyzes scientific and policy issues concerning the safety and effectiveness of medical products.
Our research center opposes the pilot as proposed, but we have suggestions that would improve it. Collaboration between sponsors and qualified research institutions (QRIs) could improve first-in-human (FIH) IND submissions by helping academic researchers and industry identify scientific and regulatory
issues earlier. However, closer alignment can also create bias, especially when institutions advising sponsors may receive sponsor funding; conduct the trial; operate the reviewing Institutional Review Board (IRB); or benefit from intellectual property, publications, or future partnerships. The pilot therefore
needs to be improved to preserve independent scientific judgment, ethical oversight, and FDA authority rather than prioritize speed alone.
Questions A.1 and A.2: QRI Qualifications, Independence, and Responsibilities
A key issue is that the FDA has not shown that its 30-day IND review is a major barrier. Of 1,410 initial INDs submitted to CDER in FY2013, only 125 (8.9%) were placed on clinical hold, and more than half became active within one year. Clinical holds remain a critical safeguard in first-in-human trials, where
risks may be unknown or irreversible. Moreover, most holds involved chemistry, manufacturing, and controls deficiencies that deserve additional attention and could often be addressed through existing guidance.1 To justify a new program, the FDA should publish current data on whether delays stem from
Agency review or incomplete submissions and prioritize stronger guidance, pre-IND consultation, and clearer templates. Because innovative drugs required a median of approximately 8.3 years of clinical development from 2010–20202, the FDA must show that accelerating the IND stage would meaningfully shorten development without weakening participant protections. Institutions experienced only with conventional drugs should not automatically qualify to evaluate higher-risk products such as gene, cell, or nucleic acid therapies.
To reduce conflicts of interest, it is essential that the FDA should prohibit the same QRI personnel from serving as both regulatory advisers and IRB or ethics reviewers. QRIs operating trial sites should publicly disclose financial interests and maintain strict separation among regulatory support, trial conduct, and ethics review. Sponsors may retain ownership of IND submissions, but if they alter QRI analyses, that must be made public, not suppressed. QRIs must preserve complete records and report unresolved concerns or possible data misrepresentation directly to the FDA. The FDA should receive both the sponsor’s response and the QRI’s unedited assessment.
Question B.1: Bias, Conflicts of Interest, and Participant Safeguards
Allowing sponsors to pay QRIs directly creates an inherent conflict of interest, particularly when institutions want repeat business. Financial or professional incentives may influence whether QRIs identify deficiencies, challenge sponsor assumptions, or raise concerns that could delay a trial. In FIH trials, conflicts of interest can lead institutions to minimize preclinical safety signals; endorse riskier dosing or inadequate monitoring; weaken informed consent; or delay reporting concerns. Because human effects are uncertain at this stage, such bias can expose participants to avoidable, irreversible, or even fatal harm and undermine the scientific validity of the trial.3,4
The FDA must prevent QRIs from simultaneously providing regulatory support, conducting the trial, and performing IRB or ethics review without strict functional separation and independent oversight. No recruitment, consent, enrollment, or intervention should begin before the FDA authorizes the trial. High-risk products should either be excluded from the initial pilot or evaluated under additional modality-specific safeguards. The FDA should require transparent and standardized fees; include smaller, nonprofit, and academic sponsors; and ensure the pilot does not favor well-resourced companies or divert resources from the standard IND pathway.
Questions B.2 and B.3: Accountability, Transparency, and Evaluation
Since QRIs are a new category used for this pilot rather than an independently defined regulatory entity, the FDA should clarify legal responsibility among sponsors, QRIs, investigators, IRBs, and the FDA. The Agency should also delineate data custody, record-retention duties, the status of QRI-generated materials as agency records, Freedom of Information Act applicability, and protections for confidential commercial information.
Because QRIs may receive sponsor funding and provide only preliminary advice, the FDA must retain exclusive authority to determine whether an IND may proceed, require modification, or be placed on clinical hold. This preserves consistent, independent safety standards and ensures that decisions involving FIH exposure remain with the publicly accountable regulator. QRI recommendations should remain advisory and must not limit the FDA’s independent review, while IRBs continue to provide separate ethical oversight and participant protections. QRIs should face corrective action for a major deficiency; and face suspension or removal for repeated failures to identify major deficiencies, undisclosed conflicts, inadequate documentation, data-integrity concerns, or suppression of scientific analyses. The FDA should publicly report review timelines; clinical holds and major or missed deficiencies; FDA-QRI disagreements; serious adverse events; participant demographics and subgroup data; and sponsor size, fees, and financial relationships. If implemented, the full pilot should undergo independent evaluation before it is revised or expanded.
Conclusions
NCHR urges the FDA not to implement the pilot as proposed. The Agency has not established that the current IND review period is a major cause of development delays, and the sponsor-funded QRI model introduces conflicts of interest that could weaken independent oversight at the stage when participants face the greatest uncertainty. If the FDA proceeds, it must require strict conflict-of-interest protections, independent IRB review, preservation of QRI scientific dissent, clear legal accountability, and transparent evaluation focused on safety and scientific quality. These safeguards are especially critical in first-in-human trials, where evidence is inherently limited, risks may be difficult to predict, and participants may be exposed to serious or irreversible harm. The FDA must therefore retain full and independent authority; require meaningful separation between sponsors, QRIs, investigators, and IRBs; and ensure that financial or institutional interests cannot override safety concerns or suppress scientific disagreement. A faster pathway should be judged not only by how quickly a trial begins, but by whether it protects participants, preserves data integrity, and maintains public trust. Without enforceable safeguards, transparency, and clear accountability, the proposed pilot risks accelerating uncertainty and harm rather than responsible innovation.
References:
1. Lapteva, L., & Pariser, A. R. (2016). Investigational New Drug applications: a 1-year pilot study
on rates and reasons for clinical hold. Journal of investigative medicine: the official publication of the American Federation for Clinical Research, 64(2), 376–382. https://doi.org/10.1136/jim2015-000010
2. Brown, D. G., Wobst, H. J., Kapoor, A., Kenna, L. A., & Southall, N. (2022). Clinical development times for innovative drugs. Nat Rev Drug Discov, 21(11), 793-794.
3. Dresser R. (2009). First-in-human trial participants: not a vulnerable population, but vulnerable nonetheless. The Journal of law, medicine & ethics: a journal of the American Society of Law, Medicine & Ethics, 37(1), 38–50. https://doi.org/10.1111/j.1748-720X.2009.00349.x
4. Koonrungsesomboon, N., Laothavorn, J., & Karbwang, J. (2016). Ethical considerations and challenges in first-in-human research. Translational Research, 177, 6-18. 3