1. The Regulatory Science of Regenerative Medicine Celia M. Witten, Ph.D., M.D., Director Office of Cellular, Tissue and Gene Therapies Center for Biologics Evaluation and Research Food and Drug Administration Translational Regenerative Medicine Forum 2011 Washington, D.C.

2. 2 Advancing Regulatory Knowledge  Guidance Documents  Outreach OCTGT Learn Webinar Series http://www.fda.gov/BiologicsBloodVaccines/NewsEvent s/ucm232821.htm http://www.fda.gov/BiologicsBloodVaccines/NewsEvent s/ucm232821.htm  Meetings Scientific meetings Meetings with individual sponsors Liaison meetings  FDA Inter-Center Collaborations FDA Commissioner’s Fellowship Program in Regenerative Medicine  Inter Agency Efforts  Advisory Committees  International Standards  Engagements with International Regulatory Authorities  Critical Path research

3. 3 Potential Benefits of Standards Use  Leverage industry efforts  Address issues not covered by FDA Guidance  Facilitate the development and maintenance of guidance  Facilitate product design  Improve time to market  Possible International harmonization

4. 4 Key Regulatory Science Questions in Regenerative Medicine  What are areas in which guidance or standards are needed and for which the science is mature enough?  What are areas in which cross cutting scientific tools are needed for product development/evaluation?

5. 5 Contact Information Celia M. Witten, Ph.D., M.D., Director Office of Cellular, Tissues and Gene Therapies CBER/FDA 1401 Rockville Pike (HFM-700) Rockville, MD 20852-1448 301-827-4163 celia.witten@fda.hhs.gov OCTGT Regulatory Issues: Patrick Riggins, Ph.D. Branch Chief, Regulatory Project Management Branch 301-827-6536 patrick.riggins@fda.hhs.gov