How do we speed up the development of life-changing therapies for rare diseases while keeping trials accessible, patient-centered, and scientifically sound?
This conference explores how decentralization, patient engagement, innovative design, and technology are reshaping rare disease trials.
Future Design and Conduct of Rare Disease Trials
Explore the next wave of trial design, including adaptive protocols, AI-driven site selection, biomarkers, and post-trial access. Learn how regulators, sponsors, and sites navigate rare disease trial complexities.
Spotlight on the Patient Experience
Dive into strategies for improving access, reducing patient burden, and designing inclusive trials. Hear how advocacy groups and patient voices shape trial frameworks and long-term engagement.
Decentralized & Hybrid Rare Disease Trials
Examine how decentralized models overcome geographic barriers, tackle recruitment challenges, and integrate home health services—all while maintaining regulatory compliance and data quality.
Leveraging Advanced Therapies & AI to Drive Breakthroughs
Wrap up with the promise of cell and gene therapies, AI, and digital biomarkers. Discuss the regulatory, ethical, and operational hurdles—and how these technologies are redefining endpoints and accelerating development.
