blank
blank

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

1:1
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
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

ECRD 2026

ECRD 2026 in Prague strengthens the push for a coordinated EU Action Plan on rare diseases

On 3–4 June, EURORDIS–Rare Diseases Europe and Orphanet convened the rare disease community at ECRD 2026 in Prague around a shared call for coordinated European action, including the forthcoming European Blueprint for Rare Diseases.
European Biotech Act 062026

ERDERA Policy Brief: European Biotech Act

The European Rare Diseases Research Alliance (ERDERA), together with the European Reference Networks (ERNs), provides the missing operational layer required to implement the European Biotech Act across the full innovation pathway —from discovery to patient access.
Female scientist in protective gear using lab equipment for research.. Photo by ima Miroshnichenko via Pexels (CC0)

Biotech Act II: European Commission opens Call for Evidence on industrial biotechnology and biomanufacturing

The European Commission has opened a Call for Evidence on the upcoming Biotech Act II — with feedback open until 10 June 2026, inviting stakeholders to submit input that will help shape the initiative before a legislative proposal is prepared.
valelopardo-doctor-1228629_1920

Evaluating public and patient involvement in interventional research–A newly developed checklist (EPPIIC)

Public and patient involvement (PPI) has been identified as an increasingly desired and, often, required component of trial methodology–leading to higher quality, more accessible and relevant clinical research.

ERDERA's

search form