PRESS RELEASE: From Principle to Practice: New Checklist Supports Patient Engagement in Rare Disease Value Research

The Center for Innovation & Value Research (the Center), a non-profit research organization committed to advancing the science, practice, and use of health technology assessment (HTA) in healthcare, is proud to announce the launch of the Rare Disease Patient Engagement (RDPE) Guidance and Checklist, a practical, downloadable resource designed to support meaningful patient and caregiver engagement across all phases of rare disease research and value assessment.

The RDPE Guidance and Checklist provides clear, actionable guidance for value researchers and their partners on collaborating with patients and caregivers in comparative effectiveness research (CER), value assessment, and economic modeling. The launch marks the completion of Phase 2 of the Center’s rare disease initiative and represents an important step toward more inclusive, patient-centered evidence generation.

“Rare disease research has long struggled with gaps between what is measured and what truly matters to patients and families,” said Rick Chapman, PhD, Head of Research at the Center. “This guidance and checklist are designed to help researchers move beyond good intentions and embed patient and caregiver perspectives directly into study design, evidence development, and decision-making. When patients are meaningfully engaged, the resulting evidence is stronger, more relevant, and more credible.”

The RDPE resource package includes:

• The RDPE Guidance and Checklist, outlining best practices and recommendations for patient engagement at each state of the research process
• Structured Checklist, available as a downloadable PDF file for real-time use and documentation
• Executive Summary, offering a high-level visual overview of the framework

Together, these tools help researchers identify data gaps, align outcomes with patient priorities, improve transparency in value assessment, and build trust-based partnerships with rare disease communities.

The RDPE Guidance and Checklist is intended for a broad audience, including value researchers, patient and caregiver communities, advocacy organizations, payers, and policy stakeholders seeking more patient-centered approaches to evidence generation.

Researchers and partners are encouraged not only to download and apply the tools but also to share real-world examples of how the checklist is being used to inform ongoing and future studies.

The Center will continue to build on this work throughout 2026 through education, partnerships, and further exploration of patient-centered methods in rare disease research and assessment.

Download the RDPE Guidance and Checklist

Conducted in partnership with the EveryLife Foundation for Rare Diseases and the National Organization for Rare Disorders ( NORD), this work is part of the Center for Innovation & Value Research’s multi-year rare disease project. It is supported by a Patient-Centered Outcomes Research Institute (PCORI) Eugene Washington Engagement Award (#EASCS-39046), with additional support from Alexion, AstraZeneca Rare Disease.

###