Waste Not, Want Not – Making the Most of Small Patient Population Data

June 24, 2021

*Recommended for Young Investigators/Researchers


Strategies for Successful Research in Small Patient Populations

  • Discuss how to support and engage in research activities and develop interest for small patient population studies

Understanding Measurement Tools to Capture Patient Data in Rare and Ultra-Rare Conditions

  • Evolutions in real-world evidence in clinical research and regulatory approaches to evidentiary standards
  • Opportunities to leverage patient-reported outcomes to further strengthen data sets

Working with Patients and Advocacy Groups to Develop Data for Disease Understanding and Clinical Development

  • Nuances of natural history study and registry data in rare diseases
  • The importance of reporting back, communicating and the individual return of results
  • Understanding potential patient and caregiver burdens in data collection strategies/tactics


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