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

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 FY2012-2016: http://www.fda.gov/downloads/aboutfda/centersoffices/cber/ucm266867.pdf CBER’s Strategic Plan for Regulatory Science and Research: http://www.fda.gov/downloads/BiologicsBloodVaccines/ScienceResearch/UCM303542.pdf

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

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

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

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

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

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)

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

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.

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

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

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

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

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

Looking Forward 2014: Moving to White Oak Campus 16

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

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!