Rare Disease Patient Engagement Guidance and Checklist (Executive Summary)
A brief overview of why this resource was developed, how it was created with multi-stakeholder input, and how it can support more patient-centered rare disease value research and HTA. DOWNLOAD
Rare Disease Patient Engagement Guidance and Checklist (Structured Checklist)
A standalone RDPE checklist table with key questions and response options organized by phase. Designed for use in team meetings, protocol reviews, and project check-ins. DOWNLOAD
Rare Disease Patient Engagement Guidance and Checklist (Guidance Text)
A detailed guide and checklist for planning, carrying out, and documenting patient and caregiver engagement. Includes relevant examples, key definitions, and HTA-relevant prompts. DOWNLOAD