Making Rare Diseases Count 2026

Unlocking impact with better data: Five strategies for success2 Stronger data systems underpin impact in rare diseases, providing the visibility, evidence and accountability needed to drive global progress. Improving data is one of the most powerful and practical ways to accelerate progress in treating rare diseases. This priority is gaining global traction, but major gaps remain. The challenge is not just to collect more data but to ensure it is meaningful and actionable.20This section sets out a roadmap built on five strategies to address critical barriers (see Figure 7). Together they provide a theory of change: better data can generate evidence of need, guide investment and drive innovation. Five strategies for smarter data collection FIGURE 7 Focus Area Challenge Solution Data collectionMany countries lack basic data Define and track a minimum dataset across countries Patient engagementPatient experiences are underweighted Strengthen patient engagement in data collection Diagnostics Many patients remain undiagnosed Improve newborn screening and diagnostic capacity Data sharingData silos and lack of interoperability Enable trusted data sharing across health systems Analytics Data is hard to collect and analyse Use AI and digital tools to address evidence gaps Source: World Economic Forum, Rare Disease Community analysis Making Rare Diseases Count: How Better Data Can Unlock a Multitrillion-Dollar Opportunity 13