FR PNH Disease Landscape

A 2025 real-world study drawn from France's national hospitalization database (PMSI, accessed via the CASD secure data platform, funded by Roche France) identified 897 PNH patients between 2018 and 2022, with the annual count rising from 581 to 725 and roughly 100 newly diagnosed patients added each year. That translates to a 2022 prevalence near 1 in 94,000, below the 1-in-70,000-to-80,000 range Orphanet and France's national rare disease plan had assumed. The same cohort shows 55.3% of patients on a C5 inhibitor, with eculizumab accounting for 89.9% of new treatment starts and ravulizumab 10.1%. This is one of the few PNH cohorts in Europe built entirely from administrative hospital records rather than a clinician-reported registry.

For commercial teams, the discrepancy is the finding: Germany's DGHO Onkopedia guideline borrows a PNH prevalence of 16 cases per million from UK and French registries, yet France's own current hospital-database figure, 897 patients against a population of roughly 68 million, near 10.6 per million, comes in lower. France also runs diagnostic infrastructure other countries reference, a French National Observatory of PNH Clones sitting on an inter-laboratory flow-cytometry harmonization program spanning more than 50 French-speaking centres since 2010. A market-sizing exercise anchored only on the literature-cited 16-per-million figure will overstate the addressable French population relative to what the country's own administrative data now shows.

This brief draws its epidemiology from France's national hospital discharge database rather than from a clinician-facing registry. The primary source is a 2025 peer-reviewed real-world study built on the PMSI, accessed through the CASD secure data platform and funded by Roche France, which tracked 897 identified PNH patients from 2018 to 2022. Where the administrative and literature estimates diverge, both figures are shown side by side rather than reconciled into a single number, since the two measure different populations.

Diagnostic-pathway detail comes from the ECLIPSE study, a French survey on time-to-diagnosis and referral patterns, and from the French National Observatory of PNH Clones, which reports on the flow-cytometry harmonization network active across more than 50 French-speaking laboratories since 2010. Reference and competence centre structure is taken from MaRIH, describing the network coordinated from Saint-Louis Hospital, AP-HP, with a pediatric centre at Robert-Debré Hospital, AP-HP.