1. Center for Biologics Evaluation and Research
Applying Regulatory Science to Advance Development of Innovative, Safe and Effective Biologic Products
Carolyn A. Wilson, Ph.D.
Associate Director for Research

2. CBER Strategic Plan for Regulatory Science Follows CBER’s Strategic Goals
Increase national preparedness to address threats from bioterrorism, pandemic and EIDs
Improve global public health through international collaboration
Enhance ability of science and technology to facilitate development of safe and effective biological products
Ensure safety of biological products
Advance regulatory science and research
Manage for organizational excellence
CBER Strategic Plan FY :
CBER’s Strategic Plan for Regulatory Science and Research:

3. Using Science and Regulation to Advance Product Development
Novel Product
Regulatory
Challenge
Public Health
Science
Discovery
New Tools
Policy/Decision
Licensed
Product
Improved
Data –
Benefit/Risk +
Opportunity to use WO move to re-engage scientific staff in the mission of CBER and that includes the critical role that research plays to support our regulatory mission.
CBER research is filling gaps in scientific knowledge in order to overcome obstacles to product development 3

4. CBER researcher = “Researcher-Regulator” ~20% CBER Staff
Integration of research and review ensures
Relevance, Expertise, Timeliness, and Usability

5. CBER Advances Regulatory Science through External Collaborations
Data from FY13 CBER Research
Reporting Database

6. Annual Review of Research
PI provides
For each project
Progress report
Future plans
Budget Request
Presentations, Pubs
Other output
Information reviewed
Lab chief, DD, ADR, OD
Relevance
Productivity
Quality
Research Reporting
Database
Funding Allocated
Relevance to priority
Scientific/Reg Output
Feasiblity

7. Cyclic Peer Review of Every PI Every 4 Years
External – Site Visits
peer review by scientific experts
Internal – Promotion, Conversion, Evaluation Committee

8. Site-Visit Report
Draft report is distributed to full Advisory Committee
Final report is approved by full Advisory Committee
Final report used in many ways:
Internal peer review of research/PI by Promotion, Conversion, Evaluation Committee (PCE) for personnel actions
By PIs for improving research program
By management, resource allocation decisions may be impacted by report (pending resource availability)

9. Office of Vaccines Research and Review
Slides Courtesy of Konstantin Chumakov, PhD
Associate Director for Research, OVRR

10. Office of Vaccines Research and Review
Director
Marion Gruber, Ph.D.
Deputy Director
Philip Krause, M.D.
Associate Director for Research
Konstantin Chumakov, Ph.D.
Associate Director for Medical Policy
and Vaccine Safety
Karen Farizo, M.D.
Associate Director For
Management and Scientific Affairs
Erik Henchal, Ph.D.
Associate Director
for Regulatory Policy
Theresa Finn, Ph.D.
Division of Bacterial, Parasitic and Allergenic Products
Director: Jay Slater, M.D.
Deputy: Drusilla Burns, Ph.D.
Division of Viral Products
Director: Jerry Weir, Ph.D.
Deputy: Robin Levis, Ph.D.
Division of Vaccines and Related Products Applications
Director: Wellington Sun, Ph.D.
Deputy: Loris McVittie, Ph.D.
OVRR Mission Statement:
To protect and enhance the public health by assuring the availability of safe and effective vaccines, allergenic extracts, and other related products.

11. OVRR Regulatory Portfolio
Bacterial vaccines (inactivated) – 10
Bacterial vaccines (live attenuated) – 2
Viral vaccines (inactivated) – 10
Viral vaccines (live attenuated) – 15
Combination products (inactivated) – 4
Allergenic extracts (diagnostic and therapeutic) >2,000
Live biotherapeutic products – INDs only

12. OVRR Core Activities
Review, evaluate, and take appropriate actions on INDs, BLAs, amendments and supplements to these applications for vaccines and related products, conducting inspections, etc.
Develop policies and procedures governing the pre-market review of regulated products
Conducting research related to the development, manufacture, and evaluation of vaccines and related products

13. OVRR Regulatory Challenges
Emphasis on SAFETY:
Products for mass use (often universal)
Recipients are healthy individuals, often children
Short regulatory cycle
Seasonal influenza vaccines
Response to emerging pathogens (pandemic vaccines)
Many products are old
Innovative technologies
Research plays a critical role in regulation of vaccines

14. OVRR Research Priorities
Safety
To study factors affecting safety of regulated products, create and validate appropriate methods and standards
Efficacy
To study biomarkers and introduce new methods and standards to evaluate and improve vaccine efficacy
Availability
To develop new concepts and methods opening novel regulatory pathways to improve availability of vaccines and related products

15. Annual Program Review Cycle
Senior Investigator
Lab Chief
Division Director
Research Management Committee
Office IOD

16. Looking Forward 2014: Moving to White Oak Campus16

17. White Oak Lab Facility State-of-the-Art Vivarium
Imaging facility with MRI, digital X-ray, IVIS, ultrasound
Transgenic derivation facility
Expanded Space for Core Technologies:
Flow cytometry
Confocal microscopy
High throughput sequencing and bioinformatic support
10 BSL-3 suites
Designed to support work of at least 12 infectious agents and work of 36 PI’s
Many suites with capacity for animal holding rooms
1 suite to support sterile sorts and live cell confocal microscopy on BSL-3 agents
Insectariums (BSL-2 and BSL-3)
Suites designed to support Microarray and PCR
Expanded NMR facility and Mass Spec Suites 17

18. Your input improves CBER’s research programs
Thank you!
To the Site Visit reviewers and
Advisory Committee
Your input improves CBER’s research programs
External review is critical to fulfilling our
regulatory mission!