This ISPOR poster describes the development of the Checklist for Patient Engagement in Rare Disease Value Research, created by the Center for Innovation & Value Research to help integrate meaningful patient and caregiver engagement into comparative effectiveness research (CER) and health technology assessment (HTA). The project began with a 15-member steering committee, mutli-stakeholder discussions, and targeted literature reviews to identify gaps in current research approaches that limit how outcomes important to rare disease patients are captured.
Building on the work, a 19-member advisory board refined a systematic, research-lifecycle-aligned checklist covering four phases—Initiation & Planning, Execution, Monitoring, and Dissemination & Assessment—with structured, reflective questions (including Likert-type scales) to support assessment and accountability. The checklist is designed to be high-level yet actionable, adaptable, across diverse rare diseases, and supportive of co-creation, fair compensation, and sustained bi-directional engagement. The poster concludes that using this checklist can help identify gaps, promote continuous improvement, and strengthen patient-centered equitable value research in rare disease.