Archives: Agenda

• 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

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

• 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

• 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

• 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

• Starting on the right foot: Considerations for training and validation of AI-driven medical devices
• Clinical performance: Steps to efficient clinical validation based on case example
• Market entry: Hurdles with Notified Body review and approval
• What happens next: Iterating in the post-market setting