On new Cell Therapy products in development, how early do you recommend beginning communications with FDA and how do you begin the discussion?


There are a multitude of opportunities to interact with the FDA during the product development cycle that should be taken advantage of, with the earliest interactions beginning at the “pre-pre-IND stage”.

Pre-pre IND interactions are informal discussion, focused primarily on preclinical issues. These interactions are always handled by teleconference and there is no set time frame to schedule these interactions. In addition, they are nonbinding in nature so the FDA cannot be obligated or bound based on these discussions for any future regulatory decisions (again, these are informal interactions). Pre-pre-IND interactions were created to offer general guidance for IND-enabling preclinical programs.

Clearly, meetings with the FDA are valuable opportunities to discuss data and current and future study design and to address the development pathway to ensure that safe and effective products are brought to market in a timely manner.

One great way to begin interactions with the FDA is by utilizing a Target Product Profile, or TPP. The TPP is a useful planning tool for preclinical, IND-enabling studies and clinical trials and for effective communications with the FDA. Successful product development begins with having the end in mind, and the TPP conveys the long-term aspirational product attributes and overall intent of the development program.

For additional reading, several of my colleagues at CIRM co-authored the following paper: “Communications with the Food and Drug Administration on the Development Pathway for a Cell-Based Therapy: Why, What, When, and How?”

You can find the paper at the following link: http://stemcellstm.alphamedpress.org/content/1/11/825.long

To learn more about the TPP, please see the following link where CIRM’s Vice President of R&D, Ellen Feigal, discuss the concept in detail: http://www.cirm.ca.gov/about-stem-cells/video/target-product-profile-ellen-feigal-2011-cirm-grantee-meeting.

In addition, the following link provides the Draft Guidance from the FDA for the TPP: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm080593.pdf