Real-word evidence on healthcare resource use and associated costs in on-demand users of replacement therapies in von Willebrand disease in France: the FORvWARD study.

Fiche publication

Date publication

mars 2026

Journal

Journal of comparative effectiveness research

Auteurs

Membres identifiés du Cancéropôle Est :
Pr NERICH Virginie

Tous les auteurs :
Polack B, Nerich V, Micallef CM, Trossaërt M, Biron-Andreani C, Bornier N, Chatelanaz C, Favre-Besse F, Beoletto J, Née M, Belhassen M, Lefèvre C, Pouvourville G

Lien Pubmed

https://www.ncbi.nlm.nih.gov/pubmed/41870437

Résumé

Real-world data about use of Von Willebrand factor (VWF) concentrates to manage on-demand patients with Von Willebrand disease (VWD) are scarce. To describe and compare patients' characteristics, treatment patterns, healthcare resource use and associated costs of patients with VWD using VWF concentrates. Using the French healthcare claims database, we included adult patients with ≥1 reimbursement for a replacement therapy (RT) containing VWF concentrate between 1 January 2017 and 30 September 2021 and followed them from first RT dispensation to 31 December 2021. Treatment patterns, healthcare resource use and associated costs of RT on-demand users were evaluated over each 30-days exposure period (EP) starting the first day of each hospital stay with ≥1 RT administration. In- and out-hospital RT doses and FVIII, number of general practitioner and nurse visits, in- and out-hospital RT dispensings and length of hospitalizations and their costs were described and compared across RTs using adjusted Generalized Estimating Equation models accounting for confounding factors. Among 2540 on-demand RT users, WILFACTIN was the main RT used, followed by VONCENTO, VEYVONDI, EQWILATE and WILSTART. Overall, the mean total RT dose was 12,962 IU and the mean cost was €21,034/EP. Compared with VEYVONDI-treated EP, WILFACTIN-treated EP had significantly longer stay duration, had more out-hospital RT dose and had higher overall and in-hospital costs; VONCENTO-treated EP had more overall and in-hospital RT dose, and had higher in-hospital and RT-related costs. This first real-world study suggests that VEYVONDI seems to be a cost-saving RT compared with other RT. Future studies including clinical data should provide further evidence.