Understanding the Landscape for Rare Disease Endpoints
- New developments/approaches & trends
- Regulatory openness to complex and innovative endpoints
Measuring Burden of Illness and Quality of Life: Aligning What’s Meaningful Between Patients & Researchers
- Opportunities to improve identification of endpoints that are meaningful to patients, caregivers and researchers
- How can patients tangibly help to inform researchers on what endpoints are meaningful to them and their community?
Endpoint Implications for Access
Identifying Teachable Moments & Lessons Learned/Case Study