Pharmaceutical groups raise questions about FDA’s expedited IND pilot
Pharmaceutical industry groups argue that the US Food and Drug Administration (FDA) should provide clearer guidance on the role of qualified research institutions (QRIs) in its proposed expedited investigational new drug pilot program, which is intended to reduce the time it takes to get drug candidates into first-in-human (FIH) trials.
Under the proposed pilot, sponsors would work with QRIs to develop protocols for FIH clinical trials intended for IND submission, with the goal of generating higher quality IND submissions, which could be submitted on a rolling basis. Responding to a public docket on the pilot, commenters raised questions about how this pilot program will integrate with FDA’s existing review programs.
The pilot program was announced as part of a larger initiative—known as Operation TrialBlazer—announced in June by the Department of Health and Human Services. Officials said the effort, which includes actions by the National Institutes of Health, Advanced Research Projects Agency for Health, and the Office of the National Coordinator for Health Information Technology, is meant to accelerate clinical research and shift more clinical research to the US from overseas. (RELATED: HHS and FDA propose clinical trial reforms to expedite drug development, Regulatory Focus 23 June 2026)
FDA received more than 200 comments to the docket by the 24 August deadline.
Clarity needed on QRIs
The Alliance for Regenerative Medicine (ARM) stated that more clarity is needed regarding the role and responsibilities of the QRIs.
“The responsibilities, deliverables, and performance expectations associated with QRI participation remain underspecified in the RFI, including how QRI recommendations will be incorporated into the review process and how QRI performance will be evaluated. Greater clarity regarding roles, accountability, and expected outputs will be important for both sponsors and participating institutions,” the group wrote.
In responding to the question on the necessary capabilities, infrastructure, and leadership expertise needed to qualify for the pilot program, the Biotechnology Innovation Organization (BIO) stated that the proposal “raises important questions regarding how FDA will define, evaluate, and certify these entities, and how it will ensure they possess the appropriate expertise for the specific product type, therapeutic area, aspect of drug development (e.g., CMC, clinical, nonclinical, biostatistics, trial design), and patient population under development.”
BIO added that the success of the program “will depend on FDA’s ability to implement clear, transparent, and risk-appropriate qualification criteria that reflect both the intended role of QRIs and the diversity of development programs expected to participate.”
One question that needs to be addressed, BIO said, is whether QRIs should be formally accredited by FDA or designated by another institution. Another area that needs to be addressed is who within FDA will be responsible for making qualification determinations.
BIO suggested that FDA may want to consider requiring prospective QRIs to complete a parallel review or mock review exercise to qualify for these reviews.
The Pharmaceutical Research and Manufacturers of America (PhRMA) said FDA needs to provide clearer guidance on the QRIs with respect to selection and certification.
“To the extent the pilot employs QRIs, PhRMA recommends that FDA provide a transparent framework for the selection, certification, and ongoing oversight of QRIs. Although the Pilot Program is intended to test the utility of QRIs in supporting IND development and review, the criteria used to select and certify these organizations could have a significant impact on the effectiveness of the Program. As such, PhRMA generally encourages FDA to adopt fit-for purpose criteria that enable participation in the Pilot Program,” the lobby group wrote.
Focus on reviews
BIO said that the scope of the third-party review program should be narrower and be focused on regulatory review, rather than clinical trial site qualifications and IRB functions.
The group said it “recommends that FDA narrow the scope of QRI responsibilities to ensure the pilot is focused on its intended goal of accelerating FIH initiation. The QRI’s responsibilities should center around regulatory submission review and decoupled from clinical trial site qualification and IRB functions, which are distinct capabilities. FDA should also consider whether the envisioned QRIs will be sufficiently capable of providing the same level of expertise as FDA reviewers. Without comparable expertise, QRI-led review may add unnecessary complexity and potential inconsistency.”
Existing programs
PhRMA requested that FDA clarify how the expedited review program will interact with or enhance the existing pre-IND meeting process.
The group said that “sponsors frequently rely on pre-IND meetings to obtain FDA feedback on key aspects of a development program, including clinical trial design, nonclinical studies, and CMC information. It is not clear what QRI review will add where FDA has provided sufficient feedback through the pre-IND process and does not identify a need for additional review of IND components prior to submission. In such circumstances, involvement of a QRI could introduce additional procedural steps without necessarily improving the quality of the IND or facilitating more efficient initiation of a FIH trial.”
In response to a question related to whether additional or alternative approaches could achieve similar goals of accelerating Phase 1 FIH IND study initiation in the US, ARM stated that “the proposed pilot should complement, rather than replace, other mechanisms for accelerating FIH development.”
The group also recommended that FDA continue to explore earlier engagement with sponsors, including finding additional mechanisms that facilitate the timely resolution of scientific, manufacturing, or regulatory questions, thereby providing sponsors with greater predictability during IND development.
Added bureaucracy and fairness
In response to a question related to whether there are any risks associated with the proposed pilot program, BIO said that “the principal risk is that the pilot could add an additional layer of review rather than reduce time to safe-to-proceed, particularly if FDA must review both the QRI’s output and the underlying components. Unless QRI review is clearly decoupled from FDA’s own review, the model risks increasing workload rather than delivering efficiency gains. FDA should consider whether alternative approaches, as described elsewhere in this letter, may better address the underlying causes of delay.”
In response to a question about whether the pilot could lead to unequal access that favors well-resourced sponsors over smaller companies, ARM acknowledged this concern.
The group pointed out that “the pilot could unintentionally favor larger sponsors with greater financial resources and existing institutional relationships. We recommend FDA monitor participation across sponsor types and sizes and consider mechanisms that promote access for small biotechnology companies, emerging developers, and academic-origin programs, which may stand to benefit most from the pilot but face disproportionate barriers to entry.”
