This poster describes the co-creation of the Patient-Centered Value Research Guidance and Checklist for Rare Diseases, developed to help researchers systematically embed meaningful patient and caregiver engagement throughout comparative effectiveness research (CER) and health technology assessment (HTA). Led by the Center for Innovation & Value Research, the project combined a steering committee, multi-stakeholder discussions, targeted gray literature reviews, and an advisory board process with public comments to identify gaps and translate them into practical guidance. The resulting checklist spans four phases of research—initiation and planning, execution, monitoring, and dissemination and assessment—and includes structured, reflective questions, examples, and resources to support usability across diverse rare diseases. Validation through case studies in sickle cell disease, leukodystrophies, and generalized myasthenia gravis showed the tool’s flexibility and relevance. The poster concludes the adaptable, accessible guidance grounded in lived experience is essential for advancing patient-centered, equitable value research in rare diseases.
