Archives: Agenda
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
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
Moderator: Robert Loll, SVP, Business Development, Praxis
Lunch and networking
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
FIRESIDE CHAT: Building Patient Trust to Overcome Recruitment Barriers When an Existing Treatment Is Available
- Understanding why recruitment becomes more challenging when patients already have access to an effective treatment
- Building trust from the first conversation and helping patients and families make genuinely informed decisions
- Addressing wider recruitment barriers, including misconceptions, specialist influence and misinformation within online communities
- Involving patients in trial design to create studies that better reflect their needs, concerns and tolerance for risk
Moderator: Warren Whyte, U.S. Consulting Director, Global Data
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
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
INTERACTIVE PANEL: How to effectively leverage patient advocacy networks to strengthen clinical research
- Understanding the patient advocacy ecosystem and its growing influence on clinical research
- Leveraging patient advocacy organizations to enhance study design and patient engagement
- Learn best practice for building effective partnerships with advocacy groups to support more robust and patient-centered trials
Moderator: Alan Balch, CEO, Patient Advocate Foundation and National Patient Advocate Foundation