Simple Complexity in an Evolving World: Rising to the Challenge

beefzoologistΒιοτεχνολογία

21 Φεβ 2013 (πριν από 4 χρόνια και 3 μήνες)

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1

Overview: Division of Cellular and
Gene Therapies, Office of Cellular,
Tissue and Gene Therapies

Raj K. Puri, M.D., Ph.D.

Director, DCGT

Office of Cellular, Tissue and Gene
Therapies

FDA, CBER

CTTB Site Visit Report to CTGTAC February 10, 2012


2

Outline


Organization


Mission and Activities


Regulatory Scientist and Researcher

Reviewer Model


DCGT Research Projects




3

CBER Office of Cellular, Tissue, and Gene Therapies


(OCTGT)

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

Wilson Bryan, M.D., Director

4

Division of Cellular and Gene Therapies (DCGT)

Raj Puri, M.D., Ph.D., Division Director

Kimberly Benton, Ph.D., Deputy Director


Cellular and Tissue

Therapy Branch

Steven Bauer, Ph.D., Chief



Tumor Vaccines and

Biotechnology Branch

Raj Puri, M.D., Ph.D. Chief



Cell Therapies Branch

Keith Wonnacott, Ph.D., Chief



Gene Therapies Branch

Daniel Takefman, Ph.D.,
Chief



Gene Transfer and

Immunogenicity Branch

Andrew Byrnes, Ph.D., Chief


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OCTGT Regulated Products


Cellular therapies


Tumor vaccines and immunotherapy


Gene therapies


Tissue and tissue based products


Xenotransplantation products


Combination products


Devices used for cells/tissues


Donor screening tests (for use

with cadaveric blood samples)

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Mission


Evaluate investigational new drug, device,
biological license, and pre
-
market applications for
OCTGT products


P
rotect patients from infectious disease
transmission from tissue transplantation.


Strongly committed to partnership between FDA,
government agencies, industry, patient advocates,
scientists, and the public to promote and develop
new therapies for the 21st Century, while
protecting human subjects and maximizing
biological product safety


Plan and conduct FDA’s Mission relevant research
to support development of Cutting edge medical
products

7

OCTGT regulatory portfolio



Over 1260 active INDs and IDEs, over 5000 IND

amendments in 2011, plus consult reviews




Four 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



Enforcement actions, international activities

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DCGT Activities


Ensure the safety of cellular, gene therapy and other
products through:



Development and implementation of a comprehensive risk
-
based regulatory framework


Evaluation of new technologies for product characterization
and rapid assessment of product safety


Development of FDA Policies and Guidances for the
regulation of cellular and gene therapy and other products

9

DCGT Activities
continued..


Inspections:


of manufacturing facilities, regulatory site visits


Consultation and Education:


Provide scientific and technical advice to other CBER
Offices, FDA Centers, and Government Agencies


Information sharing and discussion with sponsors


Counterterrorism:


COOP Coordination and Laboratory Red Alert Plan


Participation in FDA’s CT exercise/simulations

10

DCGT Activities
continued..

Community Outreach: (seminars, panel
discussions, round table, workshops)



Cell Therapy including stem cell


Tumor Vaccines and Immunotherapy


Gene Therapy


Tissue Engineering


Xenotransplantation



Round Table (CIRM, JDRF, ARM)


OCTGT Learn Webinar Series


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DCGT Activities
continued..

Partnerships:


Development of Retrovirus and Adenovirus
reference material


ASTM representation and STR profiling for cell line
authentication


Workshops in collaboration with NIH “Pluripotent
Stem Cells in Translation: Early Decisions” (March
21
-
22, 2011)



MOU with NIH NINDS and NHLBI for sharing of
information and expertise; collaboration with CRM


ERCC and Fluorescence standards for microarray
and flow technologies


Inter Agency Oncology Task Force between NCI and
FDA for joint fellowship training program

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Current DCGT Research Areas


Virology


Retroviruses, adenovirus


Immunology


Immune responses to viral and plasmid vectors,


autoimmunity and immune regulation


Cell and developmental biology


Control of differentiation in animal models:


cell fate and survival, stem cell biology


Cancer biology


Molecular biomarkers, cancer vaccines, animal

models


Biotechnology


Genomics, flow cytometry, proteomics, transgenics


Microbiology of tissue safety


Pyrosequencing and whole genome sequencing


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Researcher Reviewer
Model


Cellular, tissue engineering, and gene therapies evolve
rapidly and continually present new regulatory
challenges


These novel products raise extraordinarily complex
issues


DCGT seeks to foster a cadre of Researcher
Reviewer scientists who :



perform regulatory review and identify Critical Path
research needs to enhance and promote product
development and patient safety


perform research in key areas to support the FDA
mission and help sponsors solve product
development problems to advance cellular, gene
therapy and other products to the market place


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Types of Researcher Reviewers



Principle investigators (PIs)


tenured or tenure track
researcher reviewers


Staff Scientists


tenured researcher reviewers
supporting PIs program: do both review and research


Technicians: do primarily research, some do limited
review work


Staff Fellows: do both review and research work


Postdoctoral fellows funded as ORISE: do primarily
research



Note: Resources are provided to PIs

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Responsibilities of
PI
s

Product review


INDs, IDEs, PMA, 510k, HDEs, licenses, master files,
inspections


-

regulatory mentoring by branch chiefs


Policy development



working groups, guidance development, advisory
committees

Outreach



presubmittal advice, scientific and regulatory talks,
refereeing and editing for journals, chairing sessions at
scientific conferences, scientific collaborations relevant
to the regulatory science

Research



l
ab management, training/mentoring/supervising,
publishing papers, grant writing, leveraging/collaboration

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Research
Assessment/Management


Site visit and CBER Advisory Committee
recommendations


Promotion and Conversion Evaluation (PCE)
Committee review



Regulatory workload and quality


Publications (including guidance documents,
research articles and regulatory articles)


Success in securing external funding

17

Thank you