Presentation (CTI)

OBJECTIVES: Rare diseases present significant challenges to comparative clinical effectiveness research (CER) and health technology assessment (HTA). As advancements in identifying, diagnosing, and treating these diseases accelerate, there is an increasing demand for innovative research approaches. This project builds on prior work at the Center for Innovation and Value Research, emphasizing the integration of meaningful patient engagement into rare disease value research.
METHODS: Building on recommendations for developing CER and HTA, particularly regarding patient-centered outcomes in rare diseases, we developed a checklist to systematically integrate patient engagement throughout rare disease value research. A multi-stakeholder advisory board of 20 members was convened to provide feedback, which informed the refinement of the checklist and enhanced its usability and effectiveness.
RESULTS: We developed a checklist to guide value researchers in integrating patient engagement into the processes, data, and methods of patient-centered value research in rare diseases. Feedback from the project advisory board and internal researchers highlighted key considerations for both format and context. In terms of format, they emphasized the need for high-level guidance with sufficient detail, along with rating formats (e.g., Likert scales) to support assessment. In terms of context, they highlighted the importance of bi-directional feedback mechanisms, co-creation with patients, and aligning the question flow with the research process to enhance engagement and usability.
CONCLUSIONS: This checklist underscores the importance of integrating patient engagement into the patient-centered value research process and serves as a tool for researchers and other stakeholders to systematically evaluate engagement. Moving forward, it can be used to identify gaps in rare disease research, promote continuous improvement in patient engagement, and foster a culture of accountability. By sharing this checklist, we hope to enhance rare disease value research, by emphasizing the need for ongoing assessment and improvement of patient engagement processes.