2025 Session Videos Skip to Videos All | Opening Keynotes | Trial Design | Patient Voice | Clinical Operations | Commercialization | Closing Panel | Opening Keynote: The Evolving Regulatory Environment for Rare Disease Trials Opening Keynote: The Future of Rare Disease Trials Accelerating Rare Disease Therapies: Strategies for Efficient Clinical Development Adaptive & Precision-Driven Trials: Platform and MATCH Models for Rare Disease Breakthroughs Solutions for Trial Design Challenges in Rare Disease Trials Optimizing Rare Disease Clinical Trials with Real World Data Using NLP & AI AI-Driven Patient Identification and Recruitment Operationalizing Precision Medicine in Rare Disease Trials Innovating Clinical Outcome Assessments in Rare Disease Trials: Integrating Patient-Centered Measures and Digital Health Technologies Reducing Patient Burden in Rare Disease Trials Ethics and Strategies for Returning Individual Patient Data Collected in Clinical Research Aligning Trials with Patient-Lived Experiences How to Accelerate the Adoption of Decentralized Trials for Rare Disease Indications Decentralized by Design: Operationalizing Hybrid Rare Disease Trials Beyond Biotech: The Rise of Nontraditional Sponsors in Rare Disease Trials Leveraging Patient Advocacy To Scale Rare Disease Trials How To Prevent Failure in Rare Disease Trials? Patient Recruitment and Engagement in in Global, Multisite Rare Disease Trials Rare Isn’t Small: Incorporating Operational Excellence into Global, Multisite Rare Disease Trials Optimizing CRO and Vendor Selection - Examples from the Field Beyond the Feasibility Survey: Strategic Site Selection to Accelerate Trials and Optimize Resources Purpose-Driven Metrics: Running Gene Therapy Trials with KPIs That Matter Designing Trials with the End in Mind: Building Market Access into Rare Disease Clinical Development What Matters to Payers: Conversations You Should Start During Phase 2 Real-World Evidence That Matters: A Hands-On Workshop in Building Credible Value Stories Conducting Rare Disease Clinical Trials to Support Market Access and Commercialization Patient-Centric Commercialization: Integrating Patient Voices into Market Access Strategies Case Studies in Rare Disease Commercialization: Lessons Learned Closing Panel: Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials
