Moving a promising drug candidate from the laboratory into its first human study can require years of preparation. The US Food and Drug Administration (FDA) is exploring a new approach designed to remove avoidable delays from that process while maintaining protections for clinical trial participants.
The proposed Expedited Investigational New Drug (IND) Pilot Program is part of Operation TrialBlazer, a broader U.S. Department of Health and Human Services initiative to modernize clinical research and accelerate drug development. FDA is currently seeking public input on the pilot, which would create a more collaborative and flexible pathway for preparing IND submissions for first-in-human Phase I trials.
Building INDs Through Earlier Collaboration
Under the proposed pilot program, sponsors could work with approved research partners, including academic medical centers and contract research organizations, as they prepare Phase I IND submissions for first-in-human trials.
A rolling IND submission platform would allow information to be provided during the pre-IND process. The FDA expects earlier collaboration to result in stronger submissions and fewer problems that could trigger a clinical hold.
The proposal is one component of the FDA’s effort to simplify early-stage regulatory processes and better align submission expectations with the development phase.
Navigating FDA Resources
As part of the pilot program, the FDA has launched a Phase I IND Navigator that brings together IND application resources, including guidance documents, procedures and policies. The centralized resource may be especially helpful for smaller companies with limited in-house regulatory support.
In addition, a new Phase I Contact Center, accessible through the IND Navigator website, gives sponsors a direct resource for early-development questions and can route more specialized inquiries to agency experts.
Clarifying What Phase I Requires
The FDA is also taking steps to clarify the information sponsors need to prepare for early clinical development. One area of focus is Chemistry, Manufacturing and Controls (CMC) Flexibilities. The agency notes that excessive CMC submissions during Phase I preparation can add work and extend development timelines. The agency estimates that more focused minimum requirements could help sponsors reduce those timelines by up to 12 months.
Supporting First-in-Human Dose Selection
Another early-stage action focuses on selecting the initial dose for first-in-human trials. The FDA has issued draft guidance on using quantitative systems pharmacology (QSP) to support dose selection using the minimum anticipated biological effect level (MABEL) approach. The guidance addresses therapies suited to the MABEL approach, particularly newer products whose mechanisms of action make initial dose selection more complex. The FDA presents the approach as another step toward reducing reliance on animal toxicology when determining initial human doses.
Other recent FDA actions have addressed similar opportunities to streamline development. Draft guidance for certain cell and gene therapies describes how developers can build on prior findings rather than repeat studies. The agency has also issued draft guidance for streamlined approaches to nonclinical safety assessments for certain oncology pharmaceuticals.
Modernization Extends to Late-Stage Trials
Operation TrialBlazer extends beyond the IND stage. For example, the FDA has revised its draft guidance to clarify the circumstances under which one “rigorous, adequate and well-controlled pivotal clinical investigation,” combined with confirmatory evidence, may be used to provide substantial evidence of effectiveness to support drug approval.
The agency has also revised its master protocol guidance to include basket trials, as well as umbrella and platform trials. Using a shared trial structure can enable evaluation of multiple questions without building separate studies and data systems for each.
Taken together, the proposed changes could help streamline drug development, reducing unnecessary delays while maintaining the evidence and safeguards needed to bring new therapies to patients.
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