OBJECTIVES: Von Willebrand disease is an encountered inherited bleeding disorder affecting males/females, causing mucous membrane, skin bleeding symptoms, surgical bleeding or other hemostatic challenges. This study compared healthcare resource use and costs (HRUC) according to the replacement treatments (RT) used in on-demand users.

METHODS: Using the French national healthcare claims database (SNDS), patients with ≥1 reimbursement for a RT between 2017/01/01 and 2021/09/30 were included and followed up to 2021/12/31, loss to follow-up, or death. Using a published algorithm to identify on-demand users in the SNDS, HRUC were evaluated over 30-day assessment periods (AP), i.e. the 30-day period following the start date of each hospital stay with ≥1 RT administration. Within adult on-demand users, HRUC related to hospitalizations, in-hospital RT and FVIII, outpatient general practitioner and nurse visits, out-hospital RT and FVIII dispensings were assessed by AP and compared across RTs (Wilfactin^®, Veyvondi®, Voncento^®, Eqwilate^®) using Generalized Estimating Equation models (GEE) accounting for intra-patient correlation and potential confounding factors.

RESULTS: 4,133 APs within 2,505 adult on-demand users treated with ≥1 RT were identified. Wilfactin^® was used in 79% of AP, Voncento^® in 11%, Veyvondi^® in 8.6%, Eqwilate in 1.3%. Mean total costs (21,034€/AP) were significantly higher in Wilfactin^®-treated AP (22,152€/AP; cost ratio (CR)=1.27, 95%CI [1.13-1.42]) and Voncento^®-treated AP (19,710€/AP; CR=1.21 [1.04-1.41]) than in Veyvondi^®-treated AP (13,332€/AP). Across RTs, these costs were mainly driven by in-hospital costs (18,675€/AP). Mean RT-related costs represented 61% of mean total costs: those of Veyvondi® (9,102€/AP) were significantly lower than Voncento^® ones (14,536€/AP, CR=1,29, [1.07-1.55]), and were numerically lower to those of Wilfactin^® (12,996.0€/AP, CR=1.14 [0.97-1.33]).

CONCLUSIONS: This large-scale real-world study is the first comparing costs related to the use of RT available in France. Costs were higher in AP treated with Wilfactin^® or Voncento^® than in those treated with Veyvondi^®.