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.

<b>Background:</b> Real-world data about use of Von Willebrand factor (VWF) concentrates to manage on-demand patients with Von Willebrand disease (VWD) are scarce. <b>Aim:</b> To describe and compare patients' characteristics, treatment patterns, healthcare resource use and associated costs of patients with VWD using VWF concentrates. <b>Materials & methods:</b> 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. <b>Results:</b> 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. <b>Conclusion:</b> 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.