center for biologics evaluation and research carolyn a. wilson, ph.d. associate director for...

Center for Biologics Evaluation and

Research

Carolyn A. Wilson, Ph.D.Associate Director for Research

Applying Regulatory Science to Advance Development of

Innovative, Safe and Effective Biologic Products

CBER Our Vision

Innovative Technology AdvancingPublic 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 Regulates Complex Products

XenotransplantationXenotransplantationProductsProducts

TissuesTissues

Cell & Gene Cell & Gene TherapiesTherapies

Blood, Blood Blood, Blood Components and Components and DerivativesDerivatives

Vaccines: Preventive & Vaccines: Preventive & TherapeuticTherapeutic

Related Related DevicesDevices Allergenic ProductsAllergenic Products

Therapeutic Therapeutic ProbioticsProbiotics

CBER 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 Advance regulatory science and research Manage for organizational excellence

CBER Strategic Plan FY2012-2016:

http://www.fda.gov/downloads/aboutfda/centersoffices/cber/ucm266867.pdf

Regulatory Science:Regulatory Science:

Development and use of the scientific knowledge, tools, knowledge, tools, standards, and approachesstandards, and approaches

necessary for the assessment of medical product safety, efficacy,

quality, potency, and performance.

CBER Strategic Plan FY2012-2016:

http://www.fda.gov/downloads/aboutfda/centersoffices/cber/ucm266867.pdf

CBER Vision for Regulatory Science and Research

• Proactive, responsive, and collaborative

• Provide CBER with scientific expertise, tools, and data to support science-based decision making and policy development

Regulatory Regulatory ChallengeChallengePublic HealthPublic Health

Novel ProductNovel Product

Regulatory Regulatory ScienceScience

DiscoveryDiscoveryNew ToolsNew ToolsRegulatoryRegulatory

Policy/DecisionPolicy/Decision

Licensed Licensed ProductProduct

Improved Improved Data – Data – Benefit/RiskBenefit/Risk

+

Using Science and Regulation to Advance Product Development

CBER Organization

DirectorDeputy

Associates

Management

Compliance, Biologics Quality

Communication, Outreach and Development

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CBER Research Facilities• Biotechnology Core Facility

– Oligonucleotide, siRNA, PNA, and peptide synthesis– Peptide and DNA sequencing– Taqman Probe synthesis– HPLC; Capillary electrophoresis– Mass Spectrometry/Proteomics– Amino acid analysis

• Core support for – Flow Cytometry: Sorting/Analytic– Confocal microscopy

• Vivarium with procedure rooms– Rodents, NHP, BSL-2 capacity for infectious agents

• BSL-3 and ABSL-3 laboratories

Scientific Expertise

• Novel technologies: NMR, mass spec, flow cytometry, high throughput sequencing, microarray

• Microbiology: – parasitology, bacteriology, virology

• Immunology• Biochemistry and molecular biology• Cell and developmental biology• Biostatistics and epidemiology

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 FY11 CBER ResearchData from FY11 CBER ResearchReporting DatabaseReporting 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 VisitsExternal – Site Visits

peer review by scientific expertspeer 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)

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!