Topic: Rare Disease Project
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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
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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
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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
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2025 ISPOR Poster “Developing a Checklist for Patient Engagement in Rare Disease Value Research”
This ISPOR poster describes the development of the Checklist for Patient Engagement in Rare Disease Value Research, created by IVI to help integrate meaningful patient and caregiver engagement into comparative effectiveness research and health technology assessment.
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2025 World Orphan Drug Congress Poster “Developing a Checklist for Patient Engagement in Rare Disease Value Research”
This poster presents the development of the Checklist for Patient Engagement in Rare Disease Value Research, created by IVI to help researchers systematically integrate meaningful patient engagement into comparative effectiveness research and health technology assessment.
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2025 NORD Breakthrough Summit Poster “Co-Creating Guidance and a Checklist for Rare Disease Value Research through Meaningful Patient and Caregiver Engagement”
This poster describes the creation of the Patient-Centered Value Research Guidance and Checklist for Rare Diseases, developed to help researchers systematically embed meaningful patient and caregiver engagement through comparative effectiveness research and health technology assessment.
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2024 ISPOR Poster “Advancing Patient-Centered Outcomes Research for Rare Diseases: A Mixed-Methods Approach”
This ISPOR poster presents a mixed-methods literature review examining how patient-centered outcomes are addressed in health technology assessment and economic evaluation for rare diseases.
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2024 ISPOR Poster “Engaging Patients with Rare Disease in Identifying Meaningful Approaches to integrate Patient-Centered Outcomes inComparative Effectiveness Research and Value Assessment”
This poster summarizes a mutli-stakeholder IVI-EveryLife Foundation project focused on engaging people with rare diseases to identify meaningful, patient-centered outcomes for comparative effectiveness research and value assessment.
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Bridging the Gap, Empowering Patients: A Collaborative Approach to Rare Disease Research
This video highlights the significance of adopting a collaborative, patient-centered approach in rare disease research.
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Valuing Rare Disease Treatments in Healthcare: Real Experience, Real Impact
This report, developed by the Innovation and Value Initiative and the EveryLife Foundation for Rare Diseases, outlines how comparative effectiveness research, patient-centered outcomes research, and value assessment must evolve to better reflect what matters to people living with rare diseases and their caregivers.