Welcome to the CDER Small Business and Industry Assistance (SBIA) Podcast Series.

Today’s topic is ‘Early Engagement with FDA to Discuss Novel Surrogate Endpoints’.

In our last issue, we introduced how FDA is facilitating the use of surrogate endpoints in drug development. To continue this discussion, we would like to introduce Dr. Laura Jaeger, Science Policy Analyst for the Biomarker Development and Regulatory Science Team in FDA’s Center for Drug Evaluation and Research Office of New Drugs.

Thanks for having me. Sponsors planning to use a novel surrogate endpoint to support their product approval can now engage with FDA early in their drug development program by requesting a formal Type C IND meeting.

The purpose of this new Type C meeting is to provide sponsors with a mechanism that will help facilitate discussions with FDA about whether or not it is feasible for the sponsor to use their proposed surrogate endpoint to support accelerated or traditional approval of the therapeutic product under development. In addition, if any knowledge gaps are identified during the discussion, this Type C meeting will give both parties an opportunity to brainstorm about potential next steps that could be taken to help address outstanding issues.

The type of surrogate endpoints that would be appropriate for this new Type C meeting would include biomarkers that have never been previously used before as the primary basis for a product’s approval in the context of use that is being proposed. This category of Type C meeting is unique because the sponsor is required to submit a complete meeting background package along with their request to meet with FDA. The meeting background package should include a cover letter that clearly states the purpose of the meeting, which is to discuss a new surrogate endpoint. It should also include preliminary clinical data indicating that the drug has an impact on the surrogate endpoint and that this impact occurs at a drug dose that appears to be generally tolerable.

To facilitate this conversation, FDA has created a document summarizing the key content areas that a sponsor may want to consider addressing as part of their meeting background package. This document is available in our accompanying newsletter. It’s organized by broad topics that are broken down into a series of questions. The first section focuses on the relationship of the surrogate endpoint with the clinical outcome, the second section has questions geared towards understanding the relationship of the surrogate endpoint with the therapeutic product, and the last section considers reliability of the measurement tool or tools that were used to detect the surrogate endpoint.

As mentioned before, unlike other Type C meeting requests, a complete background package is due at the time the sponsor submits a formal request for a Type C meeting to discuss novel surrogate endpoints.  CDER strongly suggests that copies of meeting packages be provided in both electronic and paper format. The project manager will advise on the number of desk copies that are needed for meeting attendees from FDA. After a request is received, the Agency will accept or deny the meeting within 21 calendar days. If accepted, the Type C meeting will occur within 75 calendar days of the request.  Other details regarding procedural information for Type C meetings can be found in the FDA Draft Guidance: “Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products.”  

In conclusion, these Type C meetings, in combination with the Table of Surrogate Endpoints That Were the Basis of Drug Approval or Licensure, which was described in our last SBIA Chronicles podcast, are highly valuable new tools FDA is providing to assist companies that are interested in using new surrogate endpoints as part of their drug development program. Taken together, these two resources are part of FDA’s commitment to the goal of expanding existing transparency initiatives and enhancing regulatory science to facilitate drug development, which fulfills 21st Century Cures requirements and PDUFA VI Commitments.   

A link to the full SBIA Chronicles article and more information about CDER’s SBIA Program can be found at: www.fda.gov/cdersbia. Thanks for tuning in!