Center for Biologics Evaluation and Research
description
Transcript of Center for Biologics Evaluation and Research
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
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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
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!