Accelerating PKD Drug Development: Lessons from Clinical Trials, Biomarkers, & Regulatory Innovation in Rare Kidney Diseases

• Which innovations in rare kidney disease trial design are genuinely transferable to ADPKD? Focus on enrichment, trial duration, sample size, and innovative designs
• What would it take to establish a new surrogate endpoint in ADPKD? Compare the regulatory experience with proteinuria in IgAN and other glomerular diseases with the experience using TKV and eGFR in ADPKD
• Can regulatory precedents from other rare kidney diseases accelerate PKD drug development? Discuss accelerated approval, natural history evidence, patient-centered outcomes, and regulatory engagement

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