Trial Design, • 9/25/25 Operationalizing Precision Medicine in Rare Disease Trials Previous AI-Driven Patient Identification and Recruitment Next Innovating Clinical Outcome Assessments in Rare Disease Trials: Integrating Patient-Centered Measures and Digital Health Technologies You Might Also Like Real-World Evidence That Matters: A Hands-On Workshop in Building Credible Value Stories Closing Panel: Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials Adaptive & Precision-Driven Trials: Platform and MATCH Models for Rare Disease Breakthroughs Patient Recruitment and Engagement in in Global, Multisite Rare Disease Trials Opening Keynote: The Future of Rare Disease Trials
Trial Design, • 9/25/25 Operationalizing Precision Medicine in Rare Disease Trials Previous AI-Driven Patient Identification and Recruitment Next Innovating Clinical Outcome Assessments in Rare Disease Trials: Integrating Patient-Centered Measures and Digital Health Technologies You Might Also Like Real-World Evidence That Matters: A Hands-On Workshop in Building Credible Value Stories Closing Panel: Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials Adaptive & Precision-Driven Trials: Platform and MATCH Models for Rare Disease Breakthroughs Patient Recruitment and Engagement in in Global, Multisite Rare Disease Trials Opening Keynote: The Future of Rare Disease Trials
