Rare diseases (RD) research remains fragmented throughout Europe, resulting in delays in diagnosis, fewer medicines and reduced access to care. This research domain could greatly benefit from better coordination within and beyond Europe. The EU-cofunded EJP RD project (130 institutions, 35 countries) is developing an effective RD research ecosystem to address this issue. It improves the integration, effectiveness and social impact of RD research by developing, demonstrating and promoting Europe/world-wide research, and sharing clinical data, resources, procedures, knowledge and expertise. It also carries out and further establishes an efficient financial support model for all RD research types coupled with accelerated exploitation of research results for the benefit of patients.