Archives: Agenda
Interactive Speaker-Hosted Roundtable Discussions
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
Registration and refreshments
Chairperson’s closing remarks
Case Study: Discussing an approach to quality and clinical operations oversight in a rare disease trial at a small biotech
- Deciding the right level of oversight: key considerations
- Addressing communication hurdles and how to overcome them
- Supporting sites for operational improvement and inspection readiness
Afternoon refreshments and networking
Panel Discussion: Designing appropriate study protocols with limited patient numbers in rare diseases
- 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
Afternoon break, networking and prize draw
Fireside Chat: Keeping patients and caregivers at the center of rare disease clinical trials: collaborating with patients, caregivers, and nonprofits for better outcomes
- Understanding the provider-patient conversations: point-of-patient and provider discussions to shape trial design that is patient centric
- Survey design: creating questions focused on patient and caregiver needs during the trial and post-trial to keep patients engaged and enrolled leading to better outcomes
- Share your Rare Action: mention your post-session action to improve your process that includes rare patients, caregivers, and non-profits