Patient Voice, • 9/19/25 Beyond Biotech: The Rise of Nontraditional Sponsors in Rare Disease Trials Previous Decentralized by Design: Operationalizing Hybrid Rare Disease Trials Next Leveraging Patient Advocacy To Scale Rare Disease Trials You Might Also Like Solutions for Trial Design Challenges in Rare Disease Trials Purpose-Driven Metrics: Running Gene Therapy Trials with KPIs That Matter How To Prevent Failure in Rare Disease Trials? Opening Keynote: The Evolving Regulatory Environment for Rare Disease Trials Closing Panel: Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials
Patient Voice, • 9/19/25 Beyond Biotech: The Rise of Nontraditional Sponsors in Rare Disease Trials Previous Decentralized by Design: Operationalizing Hybrid Rare Disease Trials Next Leveraging Patient Advocacy To Scale Rare Disease Trials You Might Also Like Solutions for Trial Design Challenges in Rare Disease Trials Purpose-Driven Metrics: Running Gene Therapy Trials with KPIs That Matter How To Prevent Failure in Rare Disease Trials? Opening Keynote: The Evolving Regulatory Environment for Rare Disease Trials Closing Panel: Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials
