The RDPE Guidance & 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 that can be used in everyday work.

The RDPE Guidance & Checklist is designed for mixed teams involved in rare disease evidence generation and decision-making, including:

• Researchers and methodologists working on CER, economic models, and other value studies.
• Patient and caregiver partners and advocacy organizations collaborating on research and assessment projects.
• HTA bodies and payers reviewing evidence and making coverage and benefit design decisions.
• Clinicians and clinical researchers who help design and implement studies with people living with rare diseases.

The checklist follows four phases of a typical research or assessment process:

1. Initiation & Planning: Early partnership, roles and expectations, recruitment, participation supports, and capacity building.
2. Execution: Patient engagement in practice, including co-creation of materials and methods, accessible communciation, and integration of patient experience and preference data.
3. Monitoring: Tracking engagement activities over time, planning for missing data and evidence gaps, and adjusting based on feedback.
4. Dissemination & Assessment: Sharing results back with patients and communities, recoginizing contributions, communicating uncertainties, and preparing materials for HTA and payers.

For each phase, the checklist provides:

• A brief objective
• A question or prompt to guide discussion
• A response scale indicating how fully the item has been addressed
• Links to guidance text and examples for users who want more detail

Use the RDPE Guidance & Checklist as a practical companion to your existing processes—not as an extra form to complete.

• At project start, review the checklist as a team when you are defining your research question, partners, and timelines. Note which items are already in place and where changes are needed.
• At key decision points, revisit the relevant phase (e.g., Execution before recruitment launch; Monitoring before interim analyses; Dissemination & Assessment when preparing HTA or payer materials). Capture what you did, what you could not do, and why.
• Across projects, compare responses over time to see where engagement is improving and where additional support, training, or resources may be needed.