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