Can the Use of Biomarkers as Surrogate End Points Stimulate the Drug-Development Lull

Table of Contents

• Executive Summary
  • Strategic Considerations
  • Stakeholder Implications
• Biomarkers for Drug Discovery and Development
• The Search for Surrogate End Points
• Validated Biomarkers as Surrogate End Points
  • Characteristics of Validated Surrogate End Points
  • The Validation of a Biomarker
  • Sources of Error
    • Variability
    • Sample Size and Multiple Comparisons
• Stakeholder Interest in Biomarkers
  • Regulatory Bodies
  • The Diagnostics and Biopharmaceutical Industries
    • Oncology
    • Cardiovascular Disease
• Optimizing the Use of Biomarkers Through Innovative Study Designs
  • Adaptive Signature Design
  • Biomarker-Adaptive Threshold Design
  • Other Designs
• Outlook for Biomarkers as Surrogate End Points

Tables

• 1. Select U.S. FDA Critical Path Opportunities Initiated in 2006
• 2. Select Validated Genomic Biomarkers Cited in FDA-Approved Drug Labels
• 3. Key Alliances in Biomarker Discovery, Development, and Commercialization

Figures

• 1. Biomarker Continuum
• 2. Codevelopment Timelines of Drug, Biomarker, and Diagnostic Test
• 3. Multifactorial Drug/Disease Pathway