This poster presents the development of the Checklist for Patient Engagement in Rare Disease Value Research, created by the Center for Innovation & Value Research to help researchers systematically integrate meaningful patient engagement into comparative effectiveness research (CER) and health technology assessment (HTA). Building on earlier stakeholder work, the project used a steering committee, multi-stakeholder discussions, and targeted gray literature reviews to identify gaps in how rare disease outcomes are currently studied and valued.
The resulting checklist is designed as a practical, high-level tool aligned with the research lifecycle and organized into four phases—Initiation and Planning, Execution, Monitoring, and Dissemination and Assessment—with reflective questions and rating scales to assess engagement practices, such as budgeting for patient participation, co-creation with patients, and use of bi-directional feedback. Developed and refined through an advisory board process, the checklist aims to be adaptable across diverse rare diseases and to support accountability, continuous improvement, and more patient-centered, equitable value research.