Making Rare Diseases Count 2026

The European Union is prioritizing cross-border data accessibility through the European Health Data Space (EHDS). The regulation, which came into force in March 2025,37 will connect national health system records to ensure secure access to health data across Europe. Its implementation is being shaped through the Second Joint Action Towards the European Health Data Space (TEHDAS2), an EU-funded joint action that develops guidelines and technical specifications for EHDS operation and secondary use of health data. A comprehensive framework EHDS will enable electronic health records, laboratory and imaging results and other health data to be accessible for primary use across borders, allowing patients to carry their information with them when they seek care, improving continuity and avoiding unnecessary repetition of tests. For research and innovation, EHDS provides a decentralized infrastructure called HealthData@EU for accessing and analysing anonymized and pseudonymized data in secure processing environments. This opens new opportunities for pooling rare disease data across Europe, strengthening research, improving regulatory evidence and supporting the development of new healthcare technologies. Impact on rare diseases For the rare disease community, EHDS represents a major opportunity. A European Organisation for Rare Diseases (EURORDIS) survey found that 97% of people living with a rare disease are willing to share their health data for research on their own condition, and 95% would do so for other conditions.38 EHDS provides a vehicle to transform that willingness into action. EHDS also addresses persistent challenges in data use and reuse across Europe and serves as a model globally. From a health equity perspective, it has the potential to reduce disparities across member states, giving patients in smaller or less-resourced countries a more equal opportunity to benefit from data-driven advances. From regulation to implementation Although the regulation has entered into force, practical implementation is still under way, with key provisions on the secondary use of health data scheduled to apply from March 2029 as part of a phased roll-out. Ongoing initiatives including TEHDAS2 are producing the guidelines and technical specifications that will guide the EHDS operation and the secondary use of health data. These will undergo public consultation; patients, providers, regulators and innovators are invited to participate. CASE STUDY 3 The European Health Data Space and TEHDAS2 Making Rare Diseases Count: How Better Data Can Unlock a Multitrillion-Dollar Opportunity 20