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Research Article
23 March 2026

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

Authors: Benoit Polack https://orcid.org/0000-0002-1697-6249, Virginie Nerich https://orcid.org/0000-0002-0351-6040, Claire Marant Micallef https://orcid.org/0000-0002-5635-8605, Marc Trossaërt https://orcid.org/0000-0003-4072-839X, Christine Biron-Andreani, Nadège Bornier, Catherine Chatelanaz, … Show All … , Franck Favre-Besse https://orcid.org/0009-0006-9519-7759, Julien Beoletto, Mélanie Née https://orcid.org/0000-0001-7016-501X, Manon Belhassen https://orcid.org/0000-0002-9478-5464, Cinira Lefèvre https://orcid.org/0009-0005-5127-2617 [email protected], and Gérard de Pouvourville https://orcid.org/0009-0006-9519-7759Author Info & Affiliations
Volume 15, Number 4
https://doi.org/10.57264/cer-2025-0128
PDFFormats

Abstract

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

Plain language summary

What is this article about?

Von Willebrand disease (VWD) is a rare genetic disorder caused by missing or defective Von Willebrand factors (VWF), leading to increased bleeding risk. The disease presents various severity forms, from type 1 to 3, i.e., from absent or mild symptoms to severe and spontaneous bleedings episodes. VWD therapeutic management varies widely with disease severity, from abstaining therapy to complex treatments including replacement therapies (RT) containing Von Willebrand factor (VWF). They can be used on-demand (most cases) or in prophylaxis. They are delivered intravenously at hospital (in-hospital use) or dispensed through the hospital pharmacy for home use (i.e., out-hospital use).
Including patients between 2017 and 2021, the FORvWARD study describes real-life use of VWF concentrates in VWD patients treated on-demand, i.e., for acute bleeding events or prior to invasive medical act such asa surgery. It describes and compares the healthcare consumption and costs associated to the use of the five RT available in France to date: WILFACTIN®, VONCENTO®, EQWILATE®, WILSTART® and VEYVONDI®. Costs analyzed covered RT medications (in- and out-hospital), hospitalizations, general practitioner and nurse visits. The study used the data from the French national healthcare claims database.

What were the results?

Main results show that fewer RT doses were used in patients treated with VEYVONDI® than with WILFACTIN® or VONCENTO®. They also show that the overall costs were lower for patients treated with VEYVONDI®, compared with those treated with WILFACTIN® or VONCENTO®.

What do the results mean?

Future studies are needed to better account for clinical data, which were not all available in the database used in this study.

Supplementary Material

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