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 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 researchAdvance 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.

FDA Strategic Plan for Regulatory Science

http://www.fda.gov/ScienceResearch/SpecialTopics/RegulatoryScience/ucm267719.htm

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

Role of CBER Research

• CBER research occupies a well-defined critical niche at the end of product development pipeline

• CBER scientists are uniquely positioned to address scientific issues related to safety and efficacy of medicinal products

• The results remain in public domain and benefit the entire industry

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

Bio

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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

The purpose of research management

To reconcile investigator-initiated research model with the needs

dictated by FDA mission

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)

Office of Vaccines Research and ReviewSlides Courtesy of

Konstantin Chumakov, PhDAssociate Director for Research, OVRR

OVRR Mission Statement

Protect and enhance the public health by assuring the availability of safe and effective vaccines,

allergenic extracts, and other related products.

OVRR 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 Research• Contributes to regulation of vaccines and related

products by addressing scientific aspects of critical regulatory issues

• Develops and maintains a scientific base for establishing methods and standards designed to ensure the continued safety, purity, potency and effectiveness of vaccines and related products

• Recruits and maintains highly trained scientists who possess the expertise necessary for review of regulatory submissions and development of regulatory policies and guidance documents

• Provides scientific expertise and leadership to vaccine industry to facilitate the development and introduction of new vaccines and related products

OVRR Research Management Process

Research Manageme

nt Committee Division research portfolio

Lab Chief

Proposal

Division

Director

Laboratory projects

OVRR

Budget projection

Annual Research

Plan

CBER

Principal Investiga

tor

Current priorities

Site visit, Advisory committee recommendations

Regulatory

Division

Current

regulatory issues

OVRR Rating of Research Projects

• PUBLIC HEALTH SIGNIFICANCE– Public health need — dealing with high priority issue– Immediate regulatory relevance — pending applications exist– Strategic regulatory relevance — resolving general regulatory

challenge of major importance– Office/Division needs — fills a gap in the overall research program

• SCIENTIFIC MERIT– Scientific rationale– Originality and innovation– Feasibility of research approach– Budget (affordability)

• QUALIFICATIONS AND PRODUCTIVITY– Necessary qualifications– Past productivity of investigator/impact

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!