1 octgt research suzanne epstein, ph.d. associate director for research, octgt 12-14-2010

1

OCTGT Research

Suzanne Epstein, Ph.D.

Associate Director for Research, OCTGT

12-14-2010

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 DirectorCelia M.Witten, Ph.D., M.D., Director

Stephanie Simek, Ph.D. Deputy DirectorSuzanne Epstein, Ph.D. Associate Director of Research

Richard McFarland, M.D., Ph.D., Associate Director of Policy

Division of Cellular and Gene TherapiesRaj Puri, M.D., Ph.D., Director

Kimberly Benton, Ph.D., Deputy Director

Division of Human TissuesEllen Lazarus, M.D., Director

Division of Clinical Evaluation and Pharmacology/ToxicologyVacant

Wilson Bryan, M.D., Clinical Branch ChiefMercedes Serabian, MS., Pharm/Tox Branch Chief

5

OCTGT regulatory portfolio and activities

Over 1350 active INDs and IDEs, over 4500 INDamendments 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 prioritiesScientific 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)

1. 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.

2. 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.

3. 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.

4. 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.