Archives: Agenda

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: Sharing experiences and highlighting importance of decentralizing in rare disease trials

Leila Cupersmith, Rare Disease Clinical Trial Expert & Patient Advocacy

Roundtable 2: Overcoming patient retention challenges in rare disease trials

Jenifer Waldrop, Executive Director, Rare Disease Diversity Coalition

Roundtable 3: Using new technologies in rare disease trials

Bruce Bloom, Chief Collaboration Officer, Healx

• Deciding the right level of oversight: key considerations
• Addressing communication hurdles and how to overcome them
• Supporting sites for operational improvement and inspection readiness

• Considering innovative and flexible trial designs to maximise data from small populations and allow for protocol flexibility
• Realistically assessing inclusion and exclusion criteria to ensure adequate enrolment
• Minimizing patient burden throughout
• Engaging early with patient advocacy groups and regulatory agencies for protocol feedback
• Collaborating with rare disease CROs to employ statistical methods, help select appropriate endpoints and enhance trial’s chance of success