. Every AXLRx report available for the France market.
Iptacopan (Fabhalta) reached French PNH patients through AP1 — HAS granted accès précoce authorisation two weeks before the drug's EU marketing authorisation even took effect. Any new PNH entrant must be dossier-ready for this track before, not after, EU approval.
France's PMSI hospital database counts 897 PNH patients over five years; Orphanet's older estimate puts national prevalence at 850-1,000. Only 270 of them, per the manufacturer's own estimate, are eligible for a second-line oral agent.
Saint-Louis Hospital anchors France's MaRIH-coordinated PNH reference network of 14 competence centres, but only 24 of the 50-plus centres in the national clone-observatory network actively report into it. That kind of institutional structure is exactly what this workbook sizes before any individual name enters it.
HAS splits PNH into two tracks: ravulizumab holds SMR important and first-line, priced at €14,988.66 per 1100mg vial. Iptacopan cleared only ASMR III, restricted to second-line, its €29,590.96 box price negotiated against a manufacturer-declared population of 270 patients.
France's own national hospitalization database puts PNH prevalence near 1 in 94,000, 897 patients from 2018 to 2022, lower than the 16-per-million figure Germany's DGHO Onkopedia guideline borrows from French and UK registries.
France's PMSI national hospitalization database identified 897 PNH patients between 2018 and 2022, putting 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 have long cited.
HAS rated iptacopan ASMR III but restricted it to second-line use, while ravulizumab holds SMR important and first-line status. CEPS negotiated iptacopan's price against a manufacturer-estimated population of just 270 patients.
HAS restricted iptacopan to second-line use only, anemic patients with haemoglobin below 10 g/dL after at least six months on an anti-C5 inhibitor, and rated it ASMR III. Ravulizumab holds the first-line position with SMR important.