Balancing eGFR Decline & TKV Growth for Regulatory Endpoints in PKD

  • Evaluating whether TKV growth meets regulatory standards for “reasonably likely surrogate” endpoints, especially under accelerated approval pathways
  • Investigating how well eGFR decline tracks patient-centered outcomes and long-term kidney failure risk compared to TKV alone
  • Discussing whether combined endpoints (e.g. TKV + eGFR) offer stronger case for full approval versus accelerated or conditional approvals
  • Understanding global regulatory differences (FDA, EMA, PMDA etc.) in accepting imaging- and biomarker-based endpoints, and how trials can harmonize design across jurisdictions