PKD Trial Simulator: Insights from PKD Consortium as Tools for CKD Drug Development

  • Leveraging the PKD Outcomes Consortium’s joint‐model linking longitudinal total kidney volume (TKV) growth to eGFR decline or ESRD to enrich trial populations
  • Using a Clinical Trial Simulator tool to predict outcomes, design shorter or smaller trials, and optimize inclusion criteria
  • Qualifying TKV as a prognostic biomarker with FDA and EMA to enable regulatory efficiency and accelerated pathways
  • Integrating patient registry, natural history, and trial data into unified models for trial design and drug response prediction.