Center for Biologics Evaluation and Research, FDA

1
Center for Biologics Evaluation and Research, FDA

• Kathryn M. Carbone, M.D.
• Associate Director for Research

2
(No Transcript)
3
(No Transcript)
4
The Challenge

• Drug discovery and development can run as high as
  1.9 billion and is a lengthy process.
• This delays the availability of better, safer
  medical products for protecting peoples health
  and lives
• How can the CBER make biological product
  development more efficient and support better,
  safer products faster?

J. Mervis, Science, Vol 309, July 29, 2005 I
5
The Science of Biological Product Development and
Use

• 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
• FDA Critical Path Science
• FDA Critical Path Research Initiativewww.fda.gov/
  oc/initiatives/criticalpath.htm

6
(No Transcript)
7
(No Transcript)
8

• The CBER research program will be highly
  collaborative and include laboratory,
  epidemiological, statistical, and clinical
  sciences.
• 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
  quality.

9
Why CBER? Unique Role vis-à-vis the Science of
Biological 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

10
Multitasking CBER Research-Regulators

• 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
  Inspections
• Evaluation of Adverse Drug Reactions and Risk
  Assessment
• Performing research relevant to product
  evaluation of safety, efficacy, manufacturing
  Developing/evaluating scientific tools knowledge

11
FDA Critical Path ResearchInitiative CBER
Leadership

• CBER scientists can help get better products to
  patients, faster through scientific information
  obtained from
• CBER intramural research programs
• Working collaboratively with government, academic
  and industry scientists
• Extramural Science and Scientific Experts
• Critical Path Extramural grant programs planned
• All these sources used to contribute to
  Guidances, standards, and regulatory decision
  making to support product development,
  safety/efficacy assessment and review, consistent
  manufacturing

12
Applicability of CBER Intramural Research

• Its not basic vs. applied science, its how
  CBER research activity is applicable to
  supporting biologics product approvals
• Redox and adverse events from HBOC
• Methods validation for inactivation of TSE
• Hundreds of Biologics Licensing Applications and
  Investigational New Drug Applications directly
  supported by research programs (OVRR will provide
  examples)
• CBER Research Program
• Product Safety 40
• Product Quality 25
• Product Efficacy 25
• Other 10

13
Major Focus of CBER Research Program

• Creating efficient, high quality regulatory
  pathways where there are none
• Applying 21st Century science to improve
  efficiency and accuracy of established regulatory
  pathways
• Outcomes Identifying and resolving specific,
  high priority scientific challenges in product
  evaluation
• Data Quality Public evaluation of CBER Research
  through Peer reviewed journals, external Site
  Visits etc.
• Increasing CBER Research impact, including
  visibility (web site NIH catalyst workshops
  meeting representation)
• Funding these efforts (OC partnerships)
• Providing core research support Molecular tools
  and animal studies

14
CBER Research Managing Regulatory Challenges
Into Biological Product Successes

• Formal process for Internal and External Expert
  evaluation of proposed research plans
• Regulatory Priority
• Scientific quality
• Internal External evaluation of past research
  achievements
• Outcomes include Publications, Regulatory
  Policy/Guidances, Invited talks, Research QA/QC
• Internal Management reviews Yearly cycle using
  Annual Research Program Reporting
• External Site Visits Laboratory/Res-Reg/Office
  Site Visits
• Formal Peer Review for personnel retention and
  promotion

15
Innovations Research Leadership Council

• Composed of Center Leadership
• Tasked to innovate and improve processes for
• Prioritization and goal setting
• Ensuring stakeholder input into the research
  program
• Developing tools for research and regulatory
  workload assessment and evaluation of research
  applicability
• Develop Cross-cutting Research Priorities and
  Communication strategies
• Develop Center-wide scientific matrix programs
  where appropriate
• Virtual Teams coordinate scientific expertise
  across Product Offices

16
Examples of CBER Critical Path Investment
Opportunities Vaccines

• Better biochemical characterization of vaccines
  links to standardized clinical/lab outcomes
• Better methods to characterize cell substrates
  for vaccine manufacture Cell banks
• 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 adventitious agent
  inactivation for vaccines
• Enhanced clinical trial design/analysis

17
Thank you

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