The RDPE Guidance and Checklist is a practical tool to help teams plan, carry out, and document meaningful patient and caregiver engagement in rare disease value research and HTA.

It was developed through a multi-year project that included a literature review, advisory and working group meetings, rare disease focus groups, and a public comment period. The result is a flexible framework that turns core principles—equity, transparency, participation supports, and co-creation—into clear, answerable questions you can use in everyday work.