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December 2013

The Voice of Rare Disease Patients: Experiences and Expectations of over 3,000 Patients


Book coverA new EURORDIS book, The Voice of Rare Disease Patients: Experiences and Expectations of over 3,000 Patients on Rare Disease Patient Registries in Europe presents the results of an extensive consultation process including a survey of over 3,000 European patients conducted by EURORDIS as part of the EU-funded EPIRARE project to build consensus and synergies for the EU registration of rare disease patients.

Registries are instrumental tools for understanding the natural history of rare diseases, capturing unmet medical needs, gathering critical data for research, and tracking treatment benefits and risks.

The survey results show that patients understand and value a comprehensive European approach to rare disease registries, strongly favouring EU-level regulation for registries. A European registry platform should be publically funded and patients should be involved in all aspects of governance. Capacity-building is needed to ensure this patient involvement.