Impact, Challenges and Differences in Establishing Pre- Approval Patient Registries and Natural History Studies for Rare Disease Drug Development Clinical Trials 

Time: 2:15 pm
day: Clinical Operations Discussion Day


  • Registries vs natural history studies – the differences and how each contributes to the drug development process
  • Understanding the associated benefits in better describing the natural history of a rare disease to inform the drug development process
  • How to effectively incorporate multiple stakeholder perspectives when planning and designing natural history data collection efforts
  • Lessons learned from the approval of a gene therapy where a retrospective natural history study was the basis of the approval