Innovating Clinical Outcome Assessments in Rare Disease Trials: Integrating Patient-Centered Measures and Digital Health Technologies — Rare Trials Summit 2025

Trial Design

• 9/22/25

Innovating Clinical Outcome Assessments in Rare Disease Trials: Integrating Patient-Centered Measures and Digital Health Technologies

Operationalizing Precision Medicine in Rare Disease Trials

Reducing Patient Burden in Rare Disease Trials