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Center for Biologics Evaluation and Research, FDA


Center for Biologics Evaluation and Research, FDA Kathryn M. Carbone, M.D. Associate Director for Research Sponsor Perspective* Drug discovery and development can run ... – PowerPoint PPT presentation

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Title: Center for Biologics Evaluation and Research, FDA

Center for Biologics Evaluation and Research, FDA
  • Kathryn M. Carbone, M.D.
  • Associate Director for Research

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Despite Basic Medical Research Investment Going
Sponsor Perspective
  • Drug discovery and development can run as high as
    1.9 billion.
  • Lowering that number is the current Holy Grail
    of the industry.
  • How can the CBER help make biological product
    development more efficient and support more safe
    and effective products reaching the public?

J. Mervis, Science, Vol 309, July 29, 2005 I
Sponsor Perspective
  • its not the number of targets validated, or
    the number of chemicals selected. Its proof of
    concept in patients Yamada, GSKa drug is not
    a success until weve treated a patient with it.
    Fishman, Novartis
  • its still a crapshootafter 30 years in this
    business, I havent met anybody who could pick
    winners Ruffolo, Wyeth
  • But investigational products arent true winners
    until documentation of safety, efficacy and
    manufacturing adequatetoo many drugs fail too
    late in the development process.costly and

Sponsor Perspective
  • you cant manage product development
    science. But it needs to be. Ruffolo, Wyeth
  • Knowing how to maintain a healthy product
    development pipelineis more or less a matter
    of intuition. Scheller, Genentech
  • Consider the value to human health of research
    carefully managed to target the development of
    the scientific tools and knowledge needed for
    assessment of biological product safety, efficacy
    and quality manufacture
  • Critical Path Science

FDA Critical Path Research Initiative
  • Identify, focus upon and manage research to
    resolve regulatory scientific challenges to
    improve product development process and product
  • Needed to inform policy and guidance

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Why CBER? Unique Role vis-à-vis the Science of
Biologics Product Evaluation
  • Innovators create scientific tools that are
    typically applicable to their specific products
    and not shared with others in industry
  • CBER research-regulators are expert in biological
    product development AND standard scientific
    disciplines..expertise not often seen in standard
    biomedical discovery research arenas
  • CBER research-regulators see the successes,
    failures, and missed opportunities across whole
    classes of exciting and innovative products and
    want to help
  • CBER Guidance documents that are based on science
    can provide a clearer, more predictable
    regulatory path
  • CBER plays a convening and coordinating role for
    scientific needs across sponsors

Multitasking at the FDA Research Supports
Regulatory Mission
  • CBER researchers fully integrated into the
    regulatory process (50 average time)
    Researcher-Regulator model (unique to CBER)
  • Review INDs and BLAs
  • Development of Policy and Guidance Documents
  • Meeting with Sponsors and Advisory Committees
  • Participation in Pre-license and Biennial
  • Evaluation of Adverse Drug Reactions and Risk
  • Performing research relevant to product
    evaluation of safety, efficacy, manufacturing
    Developing/evaluating scientific tools knowledge

FDA Critical Path Research Initiative CBER
  • CBER scientists can help get better products to
    patients, faster through
  • CBER intramural research programs
  • Working collaboratively with government, academic
    and industry scientists
  • Critical Path Extramural grant programs
  • Guidances, standards, publications, stakeholder
    outreach, and other creative approaches to
    support product development, safety/efficacy
    assessment and review, consistent manufacturing

Applicability of Research Programs to Biologics
  • Its not basic vs. applied science, its how
    CBER research activity is applicable to
    supporting biologics product approvals
  • Hundreds of Biologics Licensing Applications and
    Investigational New Drug Applications directly
    supported by research programs (OCTGT will
    provide examples)
  • Research Programs have special relevance to
    evaluation of Biodefense Biological and Pandemic
    Influenza medical products

Major Goals for CBER Research Program
  • Creating efficient, high quality regulatory
    pathways where there are none (particularly
    important for OCTGT products)
  • Applying 21st Century science to improve
    efficiency and accuracy of established regulatory
  • Focus on Outcomes Identifying and resolving
    specific, high priority scientific challenges in
    product evaluation
  • CBER Research Program
  • Product Safety 40
  • Product Quality 25
  • Product Efficacy 25
  • Other 10

CBER Research Managing Regulatory Challenges
Into Biological Product Successes
  • Formal process for Internal and External Expert
    evaluation of proposed research plans
  • Priority
  • Scientific quality
  • Internal External evaluation of past research
  • Internal Management reviews Yearly cycle using
    Annual Research Program Reporting
  • External Site Visits Laboratory/Res-Reg/Office
    Site Visits
  • Outcomes include Publications, Regulatory
    Policy/Guidances, Invited talks, Research QA/QC

Managing to CBER Research Goals
  • Guiding Research Principles for Offices and the
  • CBER Research Leadership Council to coordinate
    development and implementation of the Principles
  • Priorities for accomplishments in the next 12
    months identified

Guiding Principles
  • The CBER research program will be highly
    collaborative and include laboratory,
    epidemiological, statistical, and clinical
  • Its scope will encompass the scientific basis of
    pre-clinical and clinical studies, manufacturing,
    regulatory submissions, inspections,
    postmarketing surveillance and Guidances.
  • The research will be of high quality, efficient,
    and directed and managed to provide outcomes that
    address scientific and regulatory challenges in
    product development, safety, efficacy and

Research Leadership Council
  • Composed of Center Scientific Leadership
  • Tasked to innovate processes for
  • Ensuring stakeholder input into the research
  • Prioritization and goal setting
  • Developing tools for regulatory workload
    assessment and evaluation of research
  • Develop Cross-cutting Research Priorities and
    Communication strategies, and Center-wide
    scientific matrix programs where appropriate

Examples of CBER Critical Path Investment
Opportunities in Cell-Tissue-Gene Therapy
  • Better characterization of cell therapies links
    to standardized clinical/lab outcomes
  • Appropriate toxicology approaches for complex
    biological products
  • New assays, standards, biomarkers, surrogates for
    complex biologics safety, efficacy and quality
  • Multipathogen and rapid detection methodologies
  • Methods validation of pathogen inactivation for
    cells, tissues and other products
  • Improving longevity/storage of cells and tissues
  • Enhanced clinical trial design/analysis

Thank you
  • To the Advisory Committee for their time,
    expertise and suggestions for continuing
    improvement of CBER research programs