Fostered Collaboration Results in Fewer Clinical Holds: A CBER OTP Pilot

An August 2025 Regulatory Focus article affirms a trend we have been experiencing at RareMoon: placing importance on early and effective communication leads to regulatory success.

The authors of the article "CBER/OTP Clinical Hold Pilot Assessment for Cellular and Gene Therapy INDs," including the respected and longtime ex-FDA colleagues Dr. Nicole Verdun, Lori Tull, and others, describe how CBER's Office of Therapeutic Products (OTP) responded to the rising volume of gene therapy INDs by launching a pilot program that focused on earlier IND-review engagement, transparency, and collaboration. This approach led to a reduction in clinical hold rates, even as the number of submissions increased rapidly.

The pilot program implemented processes such as:

• Weekly IND status meetings between FDA reviewers and discipline leadership to discuss issues identified
• Timely leadership input on challenges identified
• Bundled Information Requests
• Timely communication of potential hold issues (via information requests or teleconferences), provided earlier in the 30-day review process
• Sufficient time for the resolution of issues

The processes and results announced in this article align with our experience at RareMoon and our collaboration with CBER/OTP over the past year.

Our Experience with CBER/OTP

In the past 18 months alone, we have submitted more than a dozen advanced therapy INDs to OTP, including programs in gene regulation, cell therapies, gene editing, and other innovative modalities. Across these submissions, we have consistently seen few to no Information Requests. When they are received, they've been timely and bundled. Agency communication remains helpful, prompt, and collaborative, reflecting how a commitment to effective communication plays a significant role in advancing drug development.

Practical Strategies to De-Risk Your IND

Based on our experience, there are several practical strategies to de-risk an IND and prepare for productive, efficient interactions with the FDA. These include (but are not limited to):

• Engage regulatory and subject-matter experts early, specifically those with experience in your modality, indication, stage of development, or region. Teams familiar with the review office (such as OTP) and its expectations can provide critical insight.
• Stack SME reviews across disciplines, including nonclinical, clinical, CMC, and biostatistics, to pressure-test your data and documentation before submission.
• Maintain transparent, cross-functional communication throughout dossier preparation to ensure alignment and avoid internal disconnects.
• Engage FDA early, for example, through a well-structured Pre-IND meeting, and follow up promptly to clarify and act on any feedback received.
• Leverage Type D meetings for focused, time-sensitive discussions on specific issues, such as responding to FDA feedback or addressing key IND components.

Although our success is largely due to using the strategies mentioned, working closely with clients, careful preparation, and our dedication to clear, thorough, and well-supported submissions, these results intersect with an Agency that is actively improving its processes and adopting changes to boost communication efficiency and effectiveness.

The combination of early, purposeful dialogue, precise questions, focused touchpoints (including Type D where appropriate), and data-driven rationale creates a shared path to better, faster drug development.