Share and protect our health data: an evidence based approach to rare disease patients’ perspectives on data sharing and data protection - quantitative survey and recommendations

BACKGROUND: The needs and benefits of sharing health data to advance scientific research and improve clinical benefits have been well documented in recent years, specifically in the field of rare diseases where knowledge and expertise are limited and patient populations are geographically dispersed. Understanding what patients want and need from rare disease research and data sharing is important to ensure their participation and engagement in the process, and to ensure that these wishes and needs are embedded within research design. EURORDIS-Rare Diseases Europe regularly surveys the rare disease community to identify its perspectives and needs on a number of issues in order to represent rare disease patients and be their voice within European and International initiatives and policy developments. Here, we present key findings from a large quantitative survey conducted with patients with rare diseases and family members as part of a continuous evidence-based advocacy process developed at EURORDIS. The aim of this survey was to explore patient and family perspectives on data sharing and data protection in research and healthcare settings and develop relevant recommendations to support shaping of future data sharing initiatives in rare disease research. This survey, translated into 23 languages, was carried out via the Rare Barometer Programme and was designed to be accessible to a diverse population with a wide range of education backgrounds. It was widely disseminated via patient organisations worldwide to ensure that a wide range of voices and experiences were represented. MAIN FINDINGS: Rare disease patients, regardless of the severity of their disease and their socio-demographic profile, are clearly supportive of data sharing to foster research and improve healthcare. However, rare disease patients’ willingness to share their data does come with specific requirements in order to respect their privacy, choices and needs for information regarding the use of their data. CONCLUSIONS: To ensure sustainability and success of international data sharing initiatives in health and research for rare diseases, appropriate legislations need to be implemented and multi-stakeholder efforts need to be pursued to foster cultural and technological changes enabling the systematic integration of patients’ preferences regarding sharing of their own health data. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1186/s13023-019-1123-4) contains supplementary material, which is available to authorized users.