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June 2021

Rare disease patients’ opinion on the future of rare diseases


Report | Rare Barometer | Published June 2021

The Rare Barometer survey on the future of rare diseases collected the opinion of people living with a rare disease and their close family members on policies that may impact their lives. It has been conducted within the Rare 2030 Foresight Study, and its results have been considered in the Rare 2030 recommendations.

This online survey was open from December 2020 to January 2021. 3,998 responses were received worldwide and 3,770 in Europe.

Results show that a holistic approach to rare diseases is needed, including research, diagnosis, treatment and care, and capitalising on new technological opportunities. Such policies need to be integrated in a new European framework on rare diseases to guide National Plans for Rare Diseases and ensure that no one living with a rare disease is left behind.

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Contributors: Jessie Dubief, EURORDIS-Rare Diseases Europe (survey design, statistical analyses, report writing); Anna Kole, EURORDIS-Rare Diseases Europe (survey design); Erwan Berjonneau, EURORDIS-Rare Diseases Europe (survey design); Sandra Courbier, EURORDIS-Rare Diseases Europe (review).

Corresponding author: correspondence to Jessie Dubief, jessie.dubief@eurordis.org

Funding: Rare Barometer is a not-for-profit survey initiative run independently by EURORDIS-Rare Diseases Europe. The survey was conducted as part of the Rare2030 Foresight Study, a two-years project co-funded by the European Union Pilot Projects and Preparatory Actions Programmes (2014-2020).

Data availability: The survey data is not available in a repository as, while pseudonymised, it includes information that could make the respondents identifiable for instance by crossing the country of residence and the name of ultra-rare diseases. Collective results are available upon request to the corresponding author or to rare.barometer@eurordis.org. EURORDIS members have access to collective results for their community (e.g. all rare diseases in one country; one rare disease in Europe or worldwide; one rare disease or a group of rare diseases in one country;…) and should be contacted directly.

Cite this report: Dubief J., Kole A., Berjonneau E., Courbier S., Rare disease patients’ opinion on the future of rare diseases. A Rare Barometer survey for the Rare 2030 Foresight study. EURORDIS-Rare Diseases Europe. June 2021.

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