Archives: Agenda
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
Sharing a quickfire summary from each roundtable discussion
Lunch and networking
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
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
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