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

*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