- 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
Archives: Agenda
PANEL: Expanding rare disease trials beyond traditional geographies to access larger patient populations and reduce study start up time
- Overviewing the global trial landscape to understand opportunities and potential challenges when planning an
- international rare disease clinical trial
- Understanding how to identify potential locations and run compliant clinical trials
- Overcoming recruitment barriers when enrolling patients overseas
Lunch and networking
Reserved for event sponsor
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
Partnering with Non-Profits in Rare Disease Drug Development: Collaboration, Licensing, and Emerging Models
- Practical strategies for engaging and communicating with nonprofits to build durable partnerships and improve clinical development execution
- Approaches to fostering long-term collaboration between biopharma and nonprofit organizations
- Navigating licensing structures, IP considerations, and in-licensing opportunities involving foundations and nonprofit funders
- The expanding role of nonprofit foundations as sponsors of research and builders of therapeutic programs
Risk management in rare disease trials: Strategies for proactive planning and real-time mitigation
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
PRIZE DRAW
Visit our exhibitors’ booths throughout the day and collect stamps in order to enter our Prize Draw and be in for a chance of winning Apple devices or Amazon giftcards. The Prize Draw will take place in the Exhibition Hall. Make sure you don’t miss out!
Morning refreshments and networking
Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?
- 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