Download CBER Exective Staff Briefing

CBER Executive Staff Briefing
Status and Priorities
October 26, 2010
Robert Yetter, PhD
Associate Director for Review Management
Center for Biologics Evaluation and Research
Food and Drug Administration
CBER
Our Vision
Innovative Technology Advancing
Public Health
• Protect and improve public and individual health
in the US and globally
• Facilitate development, approval, and access to
safe and effective products and promising new
technologies
• Strengthen CBER as a pre-eminent regulatory
organization for biologics
CBER
Our Mission
To ensure the safety, purity, potency, and
effectiveness of biological products, including
vaccines, blood and blood products, and cells,
tissues, and gene therapies for the prevention,
diagnosis, and treatment of human diseases,
conditions, or injury; and help to defend the
public against the threats of emerging
infectious diseases and bioterrorism
CBER Organization
OFFICE OF THE CENTER DIRECTOR
Director: Karen Midthun, MD
Associate Director for Policy: Diane Maloney, JD
Associate Director for Review Management: Robert Yetter, PhD
Associate Director for Research: Carolyn Wilson, PhD
Associate Director for Quality Assurance: Sheryl Lard-Whiteford, PhD
Office of Vaccines Research
and Review
Director: Norman Baylor, PhD
Deputy: Marion Gruber, PhD
Office of Cellular, Tissue
and Gene Therapies
Director: Celia Witten, MD, PhD
Deputy: Stephanie Simek, PhD
Office of Blood Research
and Review
Director: Jay Epstein, MD
Deputy: Ginette Michaud, MD
Blood Applications:
Richard Davey
Hematology:
Basil Golding
Emerging & Transfusion-Transmitted Dis.:
Hira Nakhasi
Bacterial, Parasitic & Allergenic Products:
Milan Blake
Viral Products:
Jerry Weir
Vaccines & Related Product Applications:
Wellington Sun
Product Quality:
William McCormick
Office of Communication,
Outreach and Development
Director: Lorrie McNeill
Deputy: Joanne Binkley
Office of Compliance
and Biologics Quality
Director: Mary Malarkey
Deputy (Acting): Jalena Specht, JD
Divisions
Divisions
Divisions
Divisions
Divisions
Divisions
Divisions
Manufacturers Assistance
Leslie Wheelock
Disclosure and Ovresight Mgt
Susan Frantz-Bohn
Communication & Consumer Affairs
Diane Bartell
Case Management:
Robert Sausville
Manufacturing & Product Quality:
John Eltermann
Inspections & Surveillance:
Gilliam Conley
Cell & Gene Therapy:
Raj Puri
Clin. Eval. & Pharm./Tox.:
Wilson Bryan/Mercedes Serabian
Human Tissues:
Ellen Lazarus
Office of Management
Director: James Sigg
Deputy: Deirdre Hussey
Office of Biostatistics and
Epidemiology
Director: Robert Ball, MD, MPH
Deputy: Steven Anderson, PhD
Epidemiology:
Rickey Wilson
Biostatistics:
Henry Hsu
Planning, Eval & Budget
Karen O’Brien
Program Services
David Leffler
Scientific Advisors & Consultants
William Freas
Veterinary Services
Philip Snoy
Our Products
•
•
•
•
•
•
•
Blood Derivatives
Whole Blood
Blood Components
Selected Devices
Human Tissues
Vaccines (preventive and therapeutic)
Allergenic
Products
• Cell &
Gene Therapies
• Xenotransplantation products
CBER Strategic Priorities
• Enhance the nation’s public health preparedness
• Protect and promote public health globally
• Keep patients and products safe
• Improve the quality and safety of biological products
• Bring innovative products to patients
• Strengthen our human resources and performance
CBER’s 2010 Actions to Address
Key Agency Initiatives
Safety
Transparency
National and Global Public Health
Regulatory Science
Enhanced Regulatory Processes
Focus on 2011
Priorities:
Safety is Key
CBER’s Integrated Safety Teams:
Pooling our Expertise
• 3 Product-based teams
– Tissue Safety Team
– Blood Safety Team
–
Vaccine Safety Team
• With multi-disciplinary collaborations within CBER
– Product office (blood, tissue or vaccines)
– Office of Biostatistics & Epidemiology
–
Office of Compliance & Biologics Quality
–
Office of Communication, Outreach & Development
–
Office of the Center Director
CBER Safety Teams (2)
• Multi-disciplinary teams (epidemiologists,
clinical/product reviewers, compliance/manufacturing
experts, communications) to improve acquisition,
analysis, and communication of safety information
• Encompass entire product life cycle
• Use data - evaluate emerging safety issues
• Coordinate FDA response to issues with others
• Enhance collaboration with other gov’ts., WHO, etc.
• Proactive: contribute to research, policy, outreach
FDAAA Safety Activities
• Center Activities:
– Cross-Office Center group
– Part of Cross-Center Agency group
• Focus on:
– Safety Labeling Changes
– REMS Considerations
– Post-marketing Requirements
CBER Post-Marketing Safety
Collaborations
• Vaccine Adverse Event Reporting System (with CDC)
• Centers for Medicaid & Medicare Services (CMS): 45 million
enrollees
• Department of Defense
• Vaccine Safety Database (managed by CDC)
• Department of Veterans Affairs
• Indian Health Service
• Post-Licensure Rapid Immunization Safety Monitoring (linking
state immunization registries with health care database
medical records)
• International collaborations (e.g., WHO and many regulatory
and public health agencies worldwide)
2011 Priorities:
Transparency
*Overarching Agency Initiative to
Enhance Communications in All Areas
*CBER Continues to Facilitate the
Development of Safe and Effective
Biological Products
Communication to Facilitate
Product Development
• Guidance
• Workshops
• Advisory Committees
RECENT CBER GUIDANCES
Pathways for Developing
Innovative Cell & Gene Therapy Products
• Final:
– Minimally manipulated, unrelated allogeneic placental/
umbilical cord blood intended for hematopoietic reconstitution
for specified indications (10/09)
• How to pursue licensure
– Considerations for Allogeneic Pancreatic Islet Cell Products (9/09)
• Draft:
– Investigational New Drug Applications (INDs) for Minimally
Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood
Intended for Hematopoietic Reconstitution for Specified Indications
(10/09)
• Patient access to life-saving products
– Clinical Considerations for Therapeutic Cancer Vaccines (9/09)
– Somatic Cell Therapy for Cardiac Disease (3/09)
Workshops on Cellular Products
• Public Workshop on Cell and Gene Therapy Clinical
Trials in Pediatric Populations -upcoming Nov 2,
2010
• Cord Blood Licensure: A Workshop – March 8-9,
2010
• FDA/NCI Workshop: Therapeutic Cancer Vaccines;
Considerations for Early Phase Clinical Trials Based
on Lessons Learned from Phase III - October 27,
2009
Blood Safety
Guidances Addressing Agents of Emerging Infectious Diseases
•
Guidance for Industry: Nucleic Acid Testing (NAT) for Human
Immunodeficiency Virus Type 1 (HIV-1) and Hepatitis C Virus (HCV): Testing,
Product Disposition, and Donor Deferral and Reentry (May 2010)
•
Guidance for Industry: Revised Preventive Measures to Reduce the Possible
Risk of Transmission of Creutzfeldt-Jakob Disease (CJD) and Variant
Creutzfeldt-Jakob Disease (vCJD) by Blood and Blood Products (May 2010)
•
Draft Guidance for Industry: Recommendations for the Assessment of Blood
Donor Suitability, Blood Product Safety, & Preservation of the Blood Supply in
Response to Pandemic (H1N1) 2009 Virus (11/13/2009)
•
Guidance for Industry: Use of Nucleic Acid Tests to Reduce the Risk of
Transmission of West Nile Virus from Donors of Whole Blood and Blood
Components Intended for Transfusion (11/6/2009)
Blood and Tissue Safety
Workshops Addressing Emerging Infectious Diseases
& Blood Safety
Public Workshops:
• Quantitative Risk Assessment: Blood Safety and Availability
Public Workshop -May 13, 2010
• Emerging Infectious Diseases: Evaluation to Implementation
for Transfusion and Transplantation Safety –May 11 -12, 2010
• Emerging Arboviruses: Evaluating the Threat to Transfusion
and Transplantation Safety – December 14-15, 2009
Vaccines: Guidance/ Advisory
Committee Meetings
• Public Meeting on Animal Models—Essential
Elements to Address Efficacy Under the Animal
Rule; and Reopening of Comment Period
(Docket No. FDA–2009–D–0007)-upcoming
November 5, 2010
• Guidance for Industry: Characterization and
Qualification of Cell Substrates and Other Biological
Materials Used in the Production of Viral Vaccines for
Infectious Disease Indications-March 2010
• Vaccines and Related Biological Products
Advisory Committee (VRBPAC)-May 7, 2010 :
– majority of the advisory committee members expressed
support for the continued use of Rotarix and RotaTeq and
stated that the vaccine’s benefits far outweigh the theoretical
risks posed by PCV.
2011 Priorities:
Global Collaboration to Improve Public Health
• Emerging infectious diseases
• Worldwide collaboration on emerging
pubic health threats
• Countermeasures against bioterrorism
• Worldwide collaboration to address
product quality, safety, and availability
Challenge: H1N1 Pandemic
• H1N1 response central to CBER’s
international activities
– CBER agreed to be Reference NRA for WHO
vaccine prequalification for multiple H1N1
vaccines
– CBER provides technical assistance to
developing countries via WHO
– FDA continues to work with international
authorities so that data from other countries
can inform US decision-making
CBER’s WHO Engagements
• CBER serves as reference NRA [National Regulatory
Authority] for a number of WHO prequalified vaccines
• Collaborating Center (CC) for Biological Standardization
since 1998
• Large Spectrum of Activities as CC, e.g.:
–
–
–
–
–
Expert Committee on Biological Standards
Global Advisory Committee on Vaccine Safety
Advisory Committee on HIV Vaccines
Blood Regulators Network
PAHO Training on post-marketing surveillance
– Work with WHO developing countries’ networks
Regulatory Dialogue
• Confidentiality arrangements with a
number of regulatory agencies
– European Medicines Agency (EMA)
• Product “clusters” reflecting routine, on-going
dialogue; newest cluster = Blood Products (Feb 2010)
• Parallel scientific advice
• Staff exchanges in areas of vaccines and cell, tissue &
gene therapies
– Health Canada
• Sharing regulatory information on ad hoc basis
• Mutual learning on training of staff
Harmonization Efforts
• ICH (USA, Europe, Japan) 2009/10 highlights
– 1st ICH guideline on gene therapy taken up
– Progress on interface between ICH and Standards Development
Organizations
– Expansion of Global Cooperation Group to additional individual
countries
• Global Harmonization Task Force (GHTF)
– Efforts to harmonize medical device regulatory requirements &
terminology
• Asia-Pacific Economic Cooperation
– Life Sciences Innovation Forum, regulatory harmonization efforts
launched in 2009
• Pharmaceutical Inspection Cooperation Scheme
Regulatory Science
2011 Priorities:
Using Science and Regulation to
Advance Product Development
and
Improve Product Safety
The CBER Model
• CBER research contributes to regulatory policy,
risk assessments, new methods & standards,
and changes to product labeling
• CBER researchers are fully integrated into review
process
• Together with others contribute to policy
documents
• Meet with sponsors and advisory committees
• Evaluate adverse drug reactions and do risk
assessment
Influenza Program: CBER’s Role
 Develops new high-growth influenza virus reference
strains for vaccines
 Evaluates manufacturers’ influenza seed viruses before
vaccine production
 Prepares influenza virus reagents to determine potency
of influenza vaccines
 Expanded post-market ongoing safety assessment
Photo: Wikimedia Commons
Examples: CBER Research–Based
Activities to Facilitate Product Development
• Rapid influenza hemagglutinin (HA) antibody reagent
produced by recombinant techniques (no need to grow
whole virus, then purify HA to assess vaccine potency)
• Expedited influenza vaccine reagent calibration process and
development of back-up procedures to ensure availability
• New technologies to determine vaccine potency without
using antibodies – for influenza vaccines (e.g., cell culturederived, adjuvanted, DNA, recombinant protein-based,
virus-like particles)
Meeting Challenges to Advance and
Enhance Regulatory Processes
• Some Challenges: new & on-going
 New laws/regulations (e.g., FDAAA, PREA, Patient Protection
and Affordable Care Act
 Biologics Price Competition and Innovation Act of 2009“Biosimiliars” Sections 7001-7003 of the Patient Protection
and Affordable Care Act (Public Law No. 111-148)
 PDUFA V- Reauthorization; Legislative authority expires Sept
2012
 Adoption/implementation of new standards (e.g., SPL,
CDISC/SDTM and other computer standards)
 Regulatory processes for innovative new products (especially
for treatment of serious and/or rare conditions)
Meeting Challenges to Advance and
Enhance Regulatory Processes
• Some Responses
 Workgroups to address new laws, regulations
 Enhanced reviewer training
 Public Workshops and Guidance development
 Approval Pathway for Biosimilar and Interchangeable
Biological Products Public Meeting (upcoming Nov 2-3,
2010)
 http://www.fda.gov/Drugs/NewsEvents/ucm221688.htm
 Safety Reporting Rule
(9/28/10) Final Rule: Investigational New Drug Safety Reporting
Requirements for Human Drug and Biological Products and
Safety Reporting Requirements for Bioavailability and
Bioequivalance Studies in Humans
 Standards development
A Few Notable 2010 CBER Approvals
• Menveo (meningococcal conjugate vaccine)
to prevent invasive meningococcal disease
• Prevnar 13 vaccine against more pneumococcal types
a broadened vaccine to prevent invasive
pneumococcal disease in infants and young children
• Prism automated blood screening antibody assay
for automated detection of Chagas Disease
• Provenge - autologous cellular immunotherapy
for treatment of hormone refractory prostate cancer
Acknowledgments
•
•
•
•
Karen Midthun
David Cho
Joan Wilmarth Blair
Mary Pat Leary
Thank You for Your
Attention – Questions?
Resources/Contact
• Consumer Questions about Products
- Please direct all consumer questions
about biological products to
[email protected]
• Manufacturers Assistance - Please
direct all manufacturers' assistance
questions to [email protected]