Critical Path Initiative Workshop:

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• Critical Path Initiative Workshop
• OBRR Summary
• Paul A. Mied, Ph.D.
• Blood Products Advisory Committee, 82nd Meeting
• March 17, 2005

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(No Transcript)
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The Critical Path Challenge for Blood Products
Enhance Product Safety, Purity and Potency
Avoid Product Shortages Major Increased Costs
and
Critical Path opportunities exist that could
improve blood product safety, efficacy and
availability while minimizing disruptions to the
blood system
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Critical Path Initiative

• Public Workshop October 7, 2004
• From Concept to Consumer CBER Working with
  Stakeholders on Scientific Opportunities for
  Facilitating the Development of Vaccines, Blood
  and Blood Products, and Cellular, Tissue, and
  Gene Therapies

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Critical Path Initiative

• Blood and Blood Products Panel Session
• Opportunities for FDA and Industry
• COMMUNICATION
• Increase communication with Industry issue
  Guidance Documents faster
• Establish a mandate
• Develop consistency with other agencies in
  regulatory guidelines.

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Critical Path Initiative

• Blood and Blood Products Panel Session
• Opportunities for FDA and Industry
• RESEARCH DEVELOPMENT
• Set standards in advance of product development
• Help with RD in specific areas
• Help to reduce product development time.

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Critical Path Initiative

• Blood and Blood Products Panel Session
• Opportunities for FDA and Industry
• CLINICAL DEVELOPMENT
• Set objective, firm approval requirements
• Provide more guidance on structure of clinical
  trials
• Make better use of alternatives to clinical
  trials.

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Critical Path Initiative

• Blood and Blood Products Panel Session
• Opportunities for FDA and Industry
• FDA APPLICATION REVIEW
• Accelerate product reviews
• Facilitate introduction of new donor screening
  tests
• Use alternative measures to validate product or
  process changes.

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Critical Path Example of Success

• Donor Screening for West Nile Virus RNA
• Issued an alert, call to industry, guidance
• Sponsored a public workshop
• Set standards for development of minipool NAT
• Maintained a dialog with kit manufacturers
  during development

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Critical Path Example of Success (Cont.)
Donor Screening for West Nile Virus RNA

• Approved large-scale studies under IND
• Monitoring epidemiological data and the outcome
  of nationwide testing
• Developing reference materials and standards
• Animal inoculation studies are ongoing.

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Critical Path Examples of Opportunities

• Evaluation of candidate diagnostic and donor
  screening tests for transmissible spongiform
  encephalopathies (TSEs)
• Development and validation of multiplex NAT to
  detect bacteria and parasites in blood
• Nanotechnology Novel technology for blood
  screening and diagnostic use
• Development of a valid animal model to predict
  immunogenecity of factor VIII products
• Development of standards for plasma-derived
  products (e.g., Alpha 1 PI)