Ethics and regulatory guidance to address the challenges encountered in research on rare diseases in a multinational dimension: the experience of a European funded programme

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This BMC Medical Ethics article examines ethics and regulatory guidance across EJP RD-funded rare disease research projects.

The article analyses 65 projects funded by the European Joint Programme for Rare Diseases (EJP RD) to identify ethics and regulatory issues encountered by researchers and to describe the guidance provided through the programme’s Ethics Follow-up process. Most projects addressed human participants and personal data, including genetic data, biospecimens, children and non-EU country involvement. The abstract reports that EJP RD guidance supported ethics compliance, while regulatory procedures were often overlooked, highlighting a need to strengthen regulatory awareness in rare disease research.

Acces this publication

publication

BMC Medical Ethics

Year of publication

2026

Source

BMC Medical Ethics; publisher: BMC

Author

Annalisa Landi, Yanis Mimouni, Anne Demoisy, Joana Namorado, Silvia Torretta, Annunziata D’Ercole, Marta Del Alamo, Spencer Gibson, Daria Julkowska, Viviana Giannuzzi

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