Deep Dive – Contextualizing Endpoints and Therapeutic Advances in Rare Disease

June 24, 2021

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


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