Clinical and Economic Burden of Patients with Haemophilia A and B in France: Analysis of a Nationwide Claims Database

Authors: Frenzel, L; Bouée, S; Lilliu, H; Cahoreau, V; Giraud, N; Delienne, S; Fagnani, F; Cottin, J; Bureau, I; Reynaud, A; Fahfouhi, Y; Coumert, A; Beillat, M; Mammar, N; Rudant, J; Lebreton, A

Affiliations : Hôpital Necker, Paris, France. CEMKA, Bourg-la-Reine, France. Inbeeo, London, UK. CHU de Bordeaux, Bordeaux, France. Association Française des Hémophiles, Paris, France. CHU de Dijon, Dijon, France. PFIZER, Paris, France. CHU de Clermont-Ferrand, Clermont-Ferrand, France.

Publication:  Hemophilia; 2025

ABSTRACT: INTRODUCTION: There are few data on healthcare resource use and related costs of French haemophilia A (HA) and B (HB) patients. AIMS: This study aims to describe the profile of HA and HB patients, current disease management, clinical burden and costs. METHODS: Data related to haemophilia patients of all ages alive on 1/1/2022 were extracted from the nationwide French claims database (SNDS). Patients were divided into four treatment groups: on-demand or prophylaxis with or without inhibitors. Haemophilia patients were compared with a control group (ratio 1:3) matched for age, gender and region using risk ratios (RR [95% confidence interval]). The annual direct health care costs per person were estimated. RESULTS: A total of 5,577 (HA) and 1,332 (HB) patients were included (mean age: 36.4 years). Most patients were treated on-demand (HA: 72.8%; HB: 76.6%) and a few had inhibitors (HA: 3.6%; HB: 1.1%). Overall, haemophilia clinical burden was significantly higher than among controls, in particular, mortality (RR:1.42 [1.04-1.92]), work disability (RR: 2.71 [2.22-3.30]), hospitalization for major bleeding (RR:12.06 [8.67-16.80]), orthopedic surgery (RR: 2.97 [2.65-32]) and hospitalization all causes (RR: 2.44 [2.31-2.58]). This burden was more important in patients with inhibitors or treated in prophylaxis and was close for HA and HB patients. The annual per-person costs were €282,560 and €181,566 for HA and HB in prophylaxis without inhibitors, respectively. The population with inhibitors, although limited, had even much higher costs. CONCLUSION: The clinical burden and costs of haemophilia treatments may be very high especially in patients with prophylaxis and/or with inhibitors.