This poster summarizes a multi-stakeholder IVI-EveryLife Foundation project focused on engaging people with rare diseases to identify meaningful, patient-centered outcomes for comparative effectiveness research and value assessment. Guided by a 15-member steering committee, the study combined three roundtables, a ranking survey, and a literature review to build consensus on seven key themes—patient journey and time, caregiver journey, and early and ongoing engagement, data and methods, economic impacts, scientific spillover, and identifying common patient-centered outcomes for economic modeling. The poster presents prioritized, feasible recommendations, including earlier and sustained patient and caregiver engagement throughout drug development, improved and mixed-methods data collection on outcomes that matter to patients and caregivers, explicit consideration of caregiver and economic impacts, and assessment of scientific spillover. Overall, it concludes that implementing these recommendations would significantly advance patient-centered outcomes research and value assessment in rare diseases.
