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 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

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 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

• Microbiology: parasitology, bacteriology, virology

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

CBER researcher =“Researcher-Regulator”

~20% CBER Staff

Integration of research and review ensures

Relevance, Expertise, Timeliness, and Usability

CBER RESEARCH MANAGEMENT OVERVIEW

IDENTIFICATION OF

REGULATORY AND

PUBLIC HEALTH NEEDS

CBERRESEARCH PRIORITIES

OFFICE RESEARCHPLANS ANDPRIORITIES

RESEARCHPROGRAMS

EXTERNAL REVIEW AND INPUT

CBER Research Priorities*Ensure safety, efficacy and availability of biologic

products and use and development of appropriate regulatory pathways through

• Development and evaluation of methods, reagents, standards

• Evaluation, development, integration of novel scientific technologies and preclinical models for use in product regulation, including development and analysis of novel approaches that reduce, refine, or replace use of animals (3R’s)

• Facilitation of the development of new biological products for control of high priority public health threats, including pandemic influenza, emerging infectious diseases, and agents of bioterrorism.

*FY10

• Improving clinical trial design and evaluation, including adaptive design approaches

• Enhance risk management, risk assessment, and risk communication sciences

• Developing improved analytical tools and new means to access large medical databases to perform active population-based safety surveillance, including support of FDA's Sentinel Initiative.

CBER Research Priorities - continued

Cyclic Peer Review of Every PI Every 4 Years

• Internal – Promotion, Conversion, Evaluation Committee

• External – peer review by scientific experts, Site Visits

Annual Review of ResearchPI 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

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!