1. Associate Director for Research, OCTGT
OCTGT Research
Suzanne Epstein, Ph.D.
Associate Director for Research, OCTGT

2. OCTGT Mission
Facilitate development of, approval of, and access to safe and effective CTGT products

3. Products Regulated by OCTGT
Cellular therapies
Tumor vaccines and immunotherapy
Gene therapies
Tissue and tissue-based products
Xenotransplantation products
Combination products
Devices used for cells/tissues
Cord blood

4. CBER Office of Cellular, Tissue, and Gene Therapies
Office of the Director
Celia M.Witten, Ph.D., M.D., Director
Stephanie Simek, Ph.D. Deputy Director
Suzanne Epstein, Ph.D. Associate Director of Research
Richard McFarland, M.D., Ph.D., Associate Director of Policy
Division of Cellular and Gene Therapies
Raj Puri, M.D., Ph.D., Director
Kimberly Benton, Ph.D., Deputy Director
Division of Human Tissues
Ellen Lazarus, M.D., Director
Division of Clinical Evaluation and Pharmacology/Toxicology
Vacant
Wilson Bryan, M.D., Clinical Branch Chief
Mercedes Serabian, MS., Pharm/Tox Branch Chief

5. OCTGT regulatory portfolio and activities
Over 1350 active INDs and IDEs, over 4500 IND amendments in 2009, plus consult reviews
Two licensed products, a growing number of IND products in advanced development
Devices: 510ks, PMAs, HDEs
Tissue regulations
Pre-INDs, pre-pre-IND advice
Policy and guidance, advisory committee meetings
Inspections, regulatory site visits
Enforcement actions, international activities

6. Guidance Examples Potency testing for cell and gene therapies
Therapeutic cancer vaccines
Allogeneic pancreatic islet cell products
Cell therapy for cardiac disease
Cord Blood
Good Tissue Practices
International Harmonization on Genomic Biomarkers

7. Additional DCGT regulatory portfolio and activities
Partnerships: National Toxicology Program, NIH, CDC, NCI/IOTF program, NIH stem cell task force, NIST, MATES, TERMIS, ASTM
Participation in international conference on harmonization (ICH), WHO
Outreach talks at conferences, academic institutions, consumer and patient advocacy group meetings
Liaison activities with professional groups
Publication of manuscripts on regulatory topics

8. OCTGT research: Stay ahead of the curve as products and technologies evolve
For this wide spectrum, cannot have research related to each product.
If research addressed only the products and assays of today, we would already be obsolete. We must guide pre-IND work, prepare the way for anticipated products.

9. OCTGT research: Stay ahead of the curve as products and technologies evolve
For this wide spectrum, cannot have research related to each product.
If research addressed only the products and assays of today, we would already be obsolete. We must guide pre-IND work, prepare the way for anticipated products.

10. Research Strategy in OCTGT
OCTGT products are diverse and rapidly evolving. They use new regulatory paradigms, developing rather than established
Fill scientific gaps, deal with barriers
Perform studies relevant to entire product classes (sponsors study individual products)
Make results public, and thus accessible to all sponsors, to advance the entire field
Perform horizon scanning to choose priorities
Scientific areas emphasized will be covered in the Division Director summary

11. Roles of researcher-reviewer PIs
Regulatory: product/CMC review (IND, 510k, IDE, BLA), policy and guidance development, pre-application consultation, regulatory outreach, inspections, mentoring, advisory committee preparation, enforcement, international activities
Administrative: branch chief duties, emergency preparedness, CBER coordinating committees, research management
Research: including lab management, training/mentoring
Grant writing: increasing role of competitive resources
Peer review: manuscripts, grant applications, PCE
Participation in the scientific community: scientific talks, chairing conference sessions, editing

12. Investigator recruitment
Scientific gap identified with input from staff, field of expertise endorsed by Center Director's office
Open, public recruitment with search committee.
PI recruitments in DCGT in recent years:
Field: Investigator recruited from:
Organ development Jackson Lab.
Proteomics Univ. of Kansas
Retrovirology Louisiana State University
Tissue safety Armed Forces Institute of Pathology
Immunity and tolerance Scripps Research Institute
Ongoing: iPS cells ?

13. OCTGT Research Management
Announcement of regulatory/public health goals
PI annual reports address research goals and progress, describe periodic refocusing/adjustment
Analysis of productivity and alignment with OCTGT goals
Horizon scanning for gaps, to guide recruitment
Increasing role of targeted intramural funding allocated by peer review of proposals
Tracking of outside resources
Communication tools: work in progress seminars, annual report summaries. CBER expertise database being developed.

14. 2009 OCTGT Research Priorities (responsive to CBER priorities)
Chemistry, manufacturing, controls: Development and evaluation of methods and standards for improved product characterization and lot release testing, including definition of product markers predictive of safe, effective, and consistent product performance.
Pharmacology, toxicology, product rationale: Development and evaluation of preclinical/nonclinical in vitro and in vivo methods and studies informative about the safety and efficacy of OCTGT products.
Participation in CBER-, FDA-, and DHHS-wide initiatives including risk assessment, clinical trial design and monitoring, development of markers, counterterrorism, pandemic influenza preparedness, and HIV/AIDS programs, as well as OCTGT-specific initiatives in these areas.
Improvement of the microbial safety of human tissue products by development and evaluation of methods for better processing conditions, pathogen inactivation, and/or pathogen detection.

15. Thank you to the site visitors
for reviewing CBER research programs
and providing your insights.