Fireside Discussion: Navigating Global Regulatory Expectations by Understanding Considerations for Trial Design, Surrogate Endpoints & Genotype-Driven Development to Support Efficient Global Approvals

  • What should sponsors consider when designing rare kidney disease trials intended for global approval across different regulatory jurisdictions?
  • How are regulators currently thinking about surrogate endpoints and emerging biomarkers as more therapies enter development for rare kidney diseases?
  • What regulatory considerations arise when multiple trials compete for the same small patient populations or when therapies target genetically defined subgroups?