Center for Biologics Evaluation and

ResearchApplying 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.pdfCBER’s Strategic Plan for Regulatory Science and Research:http://www.fda.gov/downloads/BiologicsBloodVaccines/ScienceResearch/UCM303542.pdf

Novel Product

Regulatory ChallengePublic Health

Regulatory Science

DiscoveryNew ToolsRegulatory

Policy/Decision

Licensed Product

Improved Data – Benefit/Risk

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Using Science and Regulation to Advance Product Development

CBER researcher =“Researcher-Regulator”

~20% CBER Staff

Integration of research and review ensures

Relevance, Expertise, Timeliness, and Usability

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50

100

150

200

250

Nu

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CBER Advances Regulatory Science through External Collaborations

Data from FY13 CBER ResearchReporting Database

Annual Review of Research

PI providesFor each project

Progress reportFuture plansBudget Request

Presentations, PubsOther output

Information reviewedLab chief, DD, ADR, ODRelevanceProductivityQuality

Research Reporting Database

Funding AllocatedRelevance to priorityScientific/Reg OutputFeasiblity

Cyclic Peer Review of Every PI

Every 4 Years

External – Site Visitspeer review by scientific experts

Internal – Promotion, Conversion, Evaluation Committee

Site-Visit Reporto Draft report is distributed to full Advisory

Committeeo Final report is approved by full Advisory

Committee o Final report used in many ways:

o Internal peer review of research/PI by Promotion, Conversion, Evaluation Committee (PCE) for personnel actions

o By PIs for improving research programo By management, resource allocation

decisions may be impacted by report (pending resource availability)

Office of Vaccines Research and Review

Slides Courtesy of Konstantin Chumakov, PhDAssociate Director for Research, OVRR

Office of Vaccines Research and ReviewDirector

Marion Gruber, Ph.D. Deputy Director

Philip Krause, M.D.

Associate Director for ResearchKonstantin Chumakov, Ph.D.

Associate Director for Medical Policy and Vaccine SafetyKaren Farizo, M.D.

Associate Director For Management and Scientific Affairs

Erik Henchal, Ph.D.

Associate Directorfor Regulatory PolicyTheresa 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.

To protect and enhance the public health by assuring the availability of safe and effective

vaccines, allergenic extracts, and other related products.

OVRR Mission Statement:

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 ActivitiesReview, 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

• EfficacyTo study biomarkers and introduce new methods and standards to evaluate and improve vaccine efficacy

• AvailabilityTo develop new concepts and methods opening novel regulatory pathways to improve availability of vaccines and related products

Annual Program Review Cycle

Senior Investigat

or

Lab Chief

Division Director

Research Managem

ent Committee

Office IOD

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Looking Forward2014: Moving to White Oak

Campus

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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

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!