center for biologics evaluation and research applying regulatory science to advance development of...
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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)