Rare Disease Patient Engagement (RDPE) Guidance and Checklist

A practical tool to help rare disease value researchers and partners plan, document, and improve meaningful patient and caregiver engagement across the research lifecycle.

Decisions about rare disease treatments are often made with limited data and outcome measures that do not fully reflect what matters most to people living with these conditions. The Rare Disease Patient Engagement (RDPE) Guidance & Checklist was created to help close this gap by making it easier for patient partners, value researchers, and decision makers to work together from the start. This resource offers clear, step-by-step questions and examples to support early and ongoing patient engagement in comparative clinical effectiveness research (CER) and health technology assessment (HTA) for rare diseases.

Start with these Core Resources

These materials are designed to work together. Begin  with the executive summary for a high-level overview, then use the full guidance document and printable checklist in your day-to-day work.

RDPE Guidance & Checklist

A detailed guide and checklist for planning, carrying out, and documenting patient and caregiver engagement. Includes relevant examples, key definitions, and HTA-relevant prompts.
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Printable 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.
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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.
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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.

Share Feedback and Examples

We welcome feedback from users who apply the RDPE Guidance & Checklist in real projects. Tell us which items were most helpful and where additional clarity or examples would be useful. Share brief case examples showing how the checklist influenced study design, HTA submissions, coverage discussions, or other decisions. Suggest additional resources, templates, or training materials that would make it easier to embed patient engagement in rare disease value research.

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Partner with the Center

The Center for Innovation & Value Research is interested in collaborating with researchers, patient organizations, HTA bodies, and payers who are interested in piloting the RDPE Guidance & Checklist in active or planned rare disease projects, co-developing training and capacity-building programs for researchers and patient partners, or exploring real-world applications in value assessment, coverage policy, and benefit design. Contact us to request a briefing or discuss collaboration opportunities.

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