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

Yes
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

You might also be interested in

GAM 2026 Badge

ERDERA General Assembly sets priorities for an integrated and delivery-focused third year

ERDERA’s third General Assembly brought partners together in Coimbra to review its second year, strengthen connections across the partnership and align priorities for a more integrated and delivery-focused third year.
ERDERA 2026 Open Session

From individual cases to new treatment pathways: ERDERA Open Session focuses on ultra-rare diseases

ERDERA marked the close of its second year with an Open Session on ultra-rare diseases, bringing together European, regulatory and scientific perspectives on how innovation can reach patients.
Blueprint EU flag

European Blueprint sets out community priorities for a coordinated EU Action Plan on Rare Diseases

EURORDIS’ European Blueprint proposes 18 priority actions across six areas, offering a community-led foundation for a future EU Action Plan on Rare Diseases.
A European Blueprint for Rare Diseases

A European Blueprint for Rare Diseases: Towards an EU Action Plan for Rare Diseases

The European Blueprint sets out how Europe can build on initiatives such as ERDERA to turn existing expertise into a more coordinated future for rare diseases.

ERDERA Search