Drugs

Support, Suggestions for Expedited IND Pilot

David McFarland

— 2 min read
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The Biotechnology Innovation Organization (BIO) says it agrees with FDA that it is critical to shorten the time it takes from drug identification to first-in-human study. In a comment letter, BIO says the agency’s proposed pilot for an expedited Investigational New Drug (IND) “presents a timely opportunity to test approaches that could meaningfully reduce delays and improve predictability in early-stage clinical development while preserving safety, scientific rigor, and regulatory oversight.”

If FDA proceeds with the pilot as envisioned, BIO recommends that it consider following these core principles:

  • pursue a phased approach to establish processes, evaluate outcomes, and identify implementation challenges before expanding to more complex modalities and patient populations;
  • preserve FDA authority and clarify the Qualified Research Institution (QRI) role;
  • streamline pre-IND engagement and predictability;
  • avoid creating new bottlenecks outside FDA review;
  • define success using balanced end-to-end metrics;
  • ensure meaningful net efficiency and FDA capacity;
  • consider scalability and broader applicability; and
  • consider how fees, capacity, and eligibility criteria may affect equitable access and ensure that the standard IND pathway remains viable and appropriately supported.

In its response, Pfizer lists comments and recommendations about specific opportunities for reform:

opportunities to modernize the IND process

  • streamline IND documentation;
  • modernize investigator listing requirements;
  • reduce the burden of duplicative IND reporting;
  • leverage new approach methodologies;
  • ensure meaningful opportunities for FDA-sponsor consultation;
  • reform clinical hold practices;
  • address digital and artificial intelligence modernization of IND preparation and review;
  • transition towards a risk-based IND safety reporting framework; and
  • drive best practices through training and education,

criteria for the expedited IND pilot program

  • establish clear criteria for QRIs;
  • preserve FDA decision-making authority and scientific dialog;
  • promote QRI alignment with Institutional Review Board review;
  • maximize the utility of rolling IND submissions; and
  • establish robust outcome metrics.

Friends of Cancer Research says the proposed pilot “provides an important opportunity to test whether new approaches can meaningfully reduce the time and complexity associated with initiating first-in-human studies.” It gives its perspectives on QRIs, rolling IND review, process efficiency, and measuring pilot success.

Parexel recommends that under the pilot, FDA should establish a high-capability QRI network, implement a predictable rolling IND review process, test the pilot across the full range of products and IND complexities, manage potential conflicts of interest through targeted safeguards, and use the pilot as a catalyst to modernize both IND review processes and preclinical/chemistry, manufacturing, and controls expectations that affect the time to first-in-human studies.

Finally, Pharmaceutical Research and Manufacturers of America (PhRMA) says it strongly supports FDA efforts to accelerate the initiation of Phase 1 first-in-human trials in the U.S. It says it is unclear how the proposed use of QRIs will accelerate the start of first-in-human trials. PhRMA offers specific recommendations for:

  • designing and implementing an effective and useful pilot program;
  • risks, oversight, and evaluation; and
  • additional considerations.

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