OBJECTIVES: Von Willebrand disease (VWD) is a rare disease caused by missing/defective von Willebrand factor (VWF). VWD-patients have an increased bleeding risk and can receive replacement therapies (RT) as long-term prophylaxis (LTP-RT) and/or on-demand (OD-RT) as part of their disease management. FORvWARD is a national real-world study aiming to describe RT-treatment patterns, healthcare resource use (HRU) and related-costs in VWD-treated patients in France.

METHODS: Using a national healthcare claims database (SNDS), patients with ≥1 RT reimbursement between 2017/01/01 and 2021/09/30 were included and followed from 1^st RT evidence until 2021/12/31, loss to follow-up, or death. Based on a previously published algorithm to distinguish LTP-RT and OD-RT (using RT frequencies, delivery types, and percentage of RT days covered), HRU/related-costs (VWD-related [e.g., RTs, VWD co-treatments, VWD-hospitalizations/specialist visits/procedures], and VWD-unrelated) were analyzed for LTP-RT and OD-RT users. Descriptive statistical analyses were conducted considering all RT exposure periods (EPs) within each LTP-RT/OD-RT population.

RESULTS: From 3,354 RT-users identified, 63 were LTP-RT patients, with 116 EPs (mean: 1.8 EPs/patient, incl. switches), mean follow-up of 4.4-y, mean age of 39.8-y, 58.7% females. In means/year per patient-period, LTP-RT patient-periods received 17.8 out-hospital RT-dispensings, 349,193 RT-IUs dispensed in- and out-hospital a RT-related cost of 347,953€. Other 3,291 patients were OD-RT users, with 4,908 EPs (mean: 1.6 EPs/patient), mean follow-up of 2.6-y, mean age of 44.1-y, 55.0% females. In means/30-days patient-period, OD-RT patient-periods had a length of hospital stay of 5.9-days, 11,810 RT-IUs dispensed in- and out-hospital, with RT-related costs of 11,701€.

CONCLUSIONS: This is the first large-scale real-world study on RT patterns of use, HRU/costs by type of RT-users in a VWD-treated population in France. These findings allow the quantification of specific disease burden and associated management costs related to each RT-user type. Future comparative analyses across RTs might identify potential treatment differences.