Archives: Agenda

• What defines a successful clinical trial and how quality planning supports strong outcomes and smooth submissions
• Building effective relationships with stakeholders, recognizing the human side of clinical research and collaboration
• Anticipating challenges and information needs, including responding to unexpected questions during the review process
• Improving efficiency across the trial lifecycle support faster delivery

• Exploring ways to effectively communicate information about your trial to wider patient populations
• Learning how to target eligible patients through increased awareness
• Connecting with communities to address patient worries in trial participation

• Practical strategies to overcome regulatory and developmental roadblocks in rare disease clinical trial planning
• Designing studies for small patient populations to generate meaningful data and gain regulatory approval
• Partnering with key opinion leaders and patient advocacy groups to inform trial design and execution
• Building a clear path from early-phase development through to Phase III and commercialization

• Clinical study design and key outcomes in sporadic ALS patients
• Mechanistic insights: targeting mitochondrial dysfunction as a therapeutic strategy
• Lessons learned and implications for future rare disease trials

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

Roundtable 2: Enhancing site relationships to ensure your rare disease clinical trial runs seamlessly

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