Understanding variability in rare disease populations

  • 16 juin 2021 - 17:00
  • Online
  • Case Study: Modeling disease progression in polycystic kidney disease to inform trial design
  • Presenters: Rhoda Muse, PhD, Scientist 1, Quantitative Medicine; Klaus Romero, MD, MS, FCP, Chief Science Officer and Executive Director of Clinical Pharmacology; Jackson Burton, PhD, Executive Director, Quantitative Medicine

The Rare Disease Cures Accelerator–Data and Analytics Platform is launching a new webinar series with the goal of sharing with the community examples of the use of rare disease person-level data in drug development and regulatory decision making. The series will feature 1-hour webinars highlighting analyses that have been done in individual disease areas, how they informed drug development, and how similar approaches could be applied to common drug development issues encountered in rare diseases. After each presentation time will be allowed for discussion with a panel of quantitative and regulatory experts around why the solutions presented were informative, lessons learned and how similar approaches could be applied to related problems.


  • Start Date:16 juin 2021
  • Start Time:17:00
  • End Date:16 juin 2021
  • End Time:18:00
  • Location:Online