Archives: Agenda

• Integrating patient insights early in protocol design to streamline clinical trial processes
• Utilizing patient networks when designing effective protocols to ensure patient centricity
• Reducing the patient burden while enhancing endpoint selection in rare disease trial design

Interactive roundtable sessions offer a unique opportunity to come together with your peers to share best practice and develop solutions to critical challenges facing the industry as a whole. Each discussion will be led by a table moderator and will focus on a different challenge within rare disease clinical trials.

Roundtables are an exciting, interactive way to build your personal network and learn from the experience and expertise of others. After 45 minutes, delegates will have the opportunity to swap and choose a different table, and each roundtable will run twice.

Roundtable 1: Reducing caregiver burden and trial fatigue in rare disease studies

Amanda Moore, CEO, Angelman Syndrome Foundation

ROUNDTABLE 2 Closing the Loop with Rare Disease Participants: How Should Sponsors Communicate During and After a Clinical Trial?

Mary E. McGowan, President and Chief Executive Officer, Foundation for Sarcoidosis

Roundtable 3: Effectively tracking vendors to ensure regulatory compliance and reduce roadblocks

• Tips and tricks on how to shorten development timelines and reach first-in-human studies faster
• Identifying opportunities to compress timelines without compromising quality
• Collaborating with CROs effectively to drive operational efficiency and aid faster study start-up

• Designing development strategies that support earlier intervention and improve long-term outcomes
• Gaining practical insights into how to optimize trial design, patient recruitment, and the use of real-world endpoints
• Addressing the complexities of small, heterogeneous pediatric populations to enhance study feasibility and success
• Engaging regulators effectively and plan for how pediatric medicines are evaluated differently—especially for cell and gene therapies and earlier treatment approaches