Making Rare Diseases Count 2026

Rare disease progress depends on how effectively data can move across institutions and borders. Yet today, most information remains scattered across electronic health records, registries and other databases that rarely communicate with one another. This fragmentation limits the usability of rare disease data, causing duplication of effort and slowing innovation. A trusted data-sharing ecosystem allows information to flow securely between stakeholders while respecting privacy, ethics and sovereignty. Federated data systems offer a compelling model for making this possible.35 In such systems, datasets stay where they are generated but insights travel freely. The goal is not to centralize information but to connect it while giving patients and institutions confidence in how their data is used. Achieving this vision requires both technical and institutional alignment. On the technical side, shared standards enable data to be captured consistently and linked across systems. On the institutional side, common governance frameworks ensure that data sharing is ethical, secure and transparent. These frameworks include privacy-by-design architecture, consent management systems and clear rules for data access and secondary use. Shared standards for data quality and validation also enable regulators and health authorities to draw on one another’s evidence, reducing duplication and accelerating access to innovation. This harmonization also helps industry, academia and patient organizations to collaborate at scale, confident that their data collection efforts will be recognized and respected across systems. Regulators are increasingly drawing on trusted counterparts to streamline reviews and reduce duplication. Collaborations such as the Access Consortium – a coalition of agencies from Canada, Switzerland, Australia, Singapore and the United Kingdom – show how aligned evidentiary standards for new medicinal products, including rare disease treatments, can speed approvals while maintaining rigour. The International Coalition of Medicines Regulatory Authorities (ICMRA) likewise called for a shared toolbox to harmonize accepted endpoints and evidence standards for rare disease therapies in its 2024 Lugano workshop recommendations.36 The benefits of trusted data-sharing frameworks are wide-ranging: for policy-makers, they improve efficiencies in health administration, strengthen oversight and enable evidence-based planning; for researchers and innovators, they expand access to representative datasets and diverse populations; for patients and families, they make participation in research and care pathways more transparent and impactful.2.4 Enable trusted data sharing across health systems 19 Making Rare Diseases Count: How Better Data Can Unlock a Multitrillion-Dollar Opportunity