Archives: Agenda

• 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

• 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

Rare disease clinical trials face unique and often underappreciated risks that can disrupt timelines, data integrity, and patient engagement. Jim will discuss both overt and nuanced risk factors, offering proactive strategies to identify, mitigate, and manage challenges across the trial lifecycle for smoother, more efficient execution

• Finding innovative solutions for patient engagement
• Collaborating with advocacy groups, patient organizations and care givers to extend patient reach
• Utilizing decentralized trials with remote visits and monitoring to reduce patient burden where possible

• Considering ways to boost recruitment through investigator meetings and recruitment workshops
• Communicating effectively and working together with sites to assist in recruitment
• Sharing recruitment challenges and successes from a rare disease trial that started during Covid-19 pandemic

• Creating vendor selection criteria and processes
• Establishing shared goals and KPIs to foster trust and alignment
• Managing expectations on timelines, budgets and deliverables
• Evaluating vendor capability in finding and supporting rare disease patients
• Monitoring vendor performance, governance and communication plans

• Sharing insights on how to use AI to bring drugs to rare disease patients
• Running through step by step processes including pre IND, FDA work, organising and conducting trial
• Discussing challenges, successes and real-world experience