FR PNH Patient Flow Model

France offers a patient count few PNH markets have: a 2025 real-world study built entirely from the national hospitalization database (PMSI, accessed via the CASD secure data platform) 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 yields 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, a range implying 850 to 1,000 patients nationally. The gap between the two is not noise: PMSI counts hospitalized, administratively captured patients, while Orphanet's older estimate is literature-derived and was never checked against hospital claims data until this study. Diagnosis concentrates at 14 competence centres coordinated through Saint-Louis Hospital's AP-HP-designated reference centre under the MaRIH rare-disease network, and a separate French National Observatory of PNH Clones, running an inter-laboratory flow-cytometry harmonisation programme since 2010, has validated 126 clone-positive cases from 24 of its 50-plus participating centres.

Of the 725 patients captured in the 2022 PMSI cohort, 55.3 percent are on a C5 inhibitor, with eculizumab accounting for 89.9 percent of new treatment starts and ravulizumab 10.1 percent, a first-line preference the reverse of what dosing convenience alone would predict. Iptacopan changes that picture only at the margin. HAS rated it ASMR III, a moderate improvement, in its 5 December 2024 opinion, but restricted the rating to second-line use only: adult patients with persistent haemolytic anaemia, haemoglobin below 10 g/dL, after at least six months on a C5 inhibitor. The manufacturer's own estimate puts that second-line-eligible population at just 270 patients nationally, a fraction of the 725-patient treated cohort and smaller still against the full prevalence range. Any France-specific sizing exercise has to work from that 270-patient ceiling, not the headline prevalence number.

Every AXLRx patient flow model is built on a five-layer funnel: population, disease burden (E1), diagnosis and specialist capture (E2), treatment and biomarker eligibility (E3), market access (E4), then Year 1-3-5 projections across three scenarios. Delivered as a live Excel workbook, not a static table: 98 formulas across 8 sheets, zero hardcoded cells.

France PNH sources: the PMSI national hospitalization database (PLOS One, 2025), Orphanet and France's national rare disease plan, the French National Observatory of PNH Clones, and HAS's Commission de la Transparence 5 December 2024 opinion on iptacopan.