Generating timely, high-quality real-world evidence (RWE) in rare diseases presents unique challenges. Limited patient populations, complex diagnostic journeys, and fragmented healthcare data make it difficult to understand true healthcare resource utilisation and inform regulatory decision-making.
CF developed an innovative data linkage methodology that connects national rare disease registry data with NHS Hospital Episode Statistics to generate comprehensive real-world evidence. This approach enabled us to analyse healthcare resource utilisation patterns three years before and five years after diagnosis, providing critical insights for rare disease management.
Key findings
Our analysis revealed distinct patterns of healthcare utilisation before and after diagnosis, with costs peaking in the three months preceding diagnosis and six months following. This stratified approach to cost estimation provides crucial insights for understanding disease burden and treatment impact.
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