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European Rare Disease Advocacy Landscape Analysis 2025

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Executive Summary

Demo Pharma Ltd

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Summary Overview

This report analyzes 16 patient advocacy groups focused on rare disease and related blood cancers, evaluating their digital presence, advocacy impact, and international reach. The analysis reveals a diverse landscape of organizations with varying strengths and areas for improvement. Some groups, like Rare Disease New Zealand and the Leukemia & Lymphoma Society, exhibit robust digital engagement and broad reach, while others, such as the Weill Cornell Medicine Rare Disease Center, have a limited online presence. Advocacy efforts are central to most groups, with focuses ranging from influencing government policy to improving treatment access. Geographically, the organizations range from highly localized to international, impacting their overall reach. Language support also varies, with Rare Disease Patients Europe offering the broadest multilingual support. Several organizations are heavily invested in research, driving advancements in the field. Overall, these groups provide a wide array of support services, addressing the complex needs of patients and families. Opportunities exist for pharmaceutical companies to strategically partner with these organizations to enhance patient outreach, improve access to care, and advance research efforts.

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