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

• Running the trial: innovative approaches and overcoming challenges
• Treating with oncolytic virus intra-tumoral therapy
• Reaching clinical success: findings and future plans

• 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

• Comparing outcomes of a DCT model versus non DCT model
• Sharing benefits and lessons learned from experiences within rare disease DCTs

• 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