7 December 2023
Today, in a significant step forward for the neuromuscular disease community, the European Reference Network for Rare Neuromuscular Diseases EURO-NMD announced the launch of the first European Registry for all rare neuromuscular diseases. The initiative aims to enhance research and improve treatment outcomes for patients. A unique feature of the EURO-NMD Registry is the option to choose from different approaches for data provision, including direct entry or CSV upload, with centralised data storage, but also by connecting local systems to the EURO-NMD Registry Hub, allowing to answer federated queries without physical data transfer between the sources.
The potential for research and trial readiness for patients treated at the Healthcare Providers (HCPs) members of the EURO-NMD network will be significantly enhanced by the Registry, which will also provide a new evidence base to implement improvements in healthcare delivery in Europe. Patients seen within our network will be best placed for exposure to groundbreaking innovation in patient care and new treatments. These objectives are enhanced through collaboration among ERNs and between EURO-NMD and other neuromuscular networks worldwide.
The EURO-NMD Registry Hub will use a pseudo-anonymisation system that guarantees data privacy.
The onboarding of ClinicalCentres (HCP sites) will start from January 2024, in a sequence determined by each centre’s readiness to enter into the necessary contractual agreements and to obtain the required ethical and legal approval for sharing data with the Registry. The EURO-NMD network currently links 82 HCPs in 25 EU and EEA member states. These HCPs are recognised as expertise centres for rare NMDs and follow more than 100,000 neuromuscular disease patients annually. There is currently no unified NMD or NMD disease-specific registry in use by all HCPs.
The team managing the EURO-NMD registry aims to provide tailored support, providing the best solution for data flow between the HCP and the Registry. All HCPs will receive:
1) an Information Letter describing the onboarding procedures;
2) a Data Sharing Agreement to be signed by the legal representative of the HCP and
3) an informed consent form template explicitly developed for the EURO-NMD Registry. We will onboard ten pilot HCPs in the first year and plan to expand the roll-out of the Registry to 40 sites in 2025, with all sites integrated by 2026-2027. The Registry management team will guide and support HCPs through all stages of the onboarding process. A helpdesk has been set up and is available via email (firstname.lastname@example.org).
We are excited in anticipation of the progress that will result from this joint effort to improve patients’ diagnosis and treatment while enhancing their access to cutting-edge research. Lastly, we wish to thank our partners (Assistance Publique Hopitaux de Paris,Universitaetsklinikum Freiburg, Stichting Katholieke Universiteit – RUMC, Stichting United Parent Projects Muscular Dystrophy UPPMD the Netherlands, Stichting Duchenne Data Foundation DDF, Association Institut de Myologie et Association Française Contre les Myopathies) and their teams for their continuous support, dedication and for helping ERN EURO-NMD reach this milestone.