Cost comparison of prophylactic vs. on-demand use of a 1:1 ratio pdVWF/FVIII concentrate in type 3 von willebrand disease with severe bleeding phenotype in the United States: A payer and societal perspective

Abstract Background Long-term prophylaxis with von Willebrand factor (VWF) concentrate is recommended for patients with von Willebrand disease (VWD) who experience frequent and severe bleeding episodes. However, few cost-effectiveness or cost-comparison studies have evaluated prophylaxis versus on-demand (OD) treatment with VWF concentrates, and most rely on assumptions or literature-based inputs. This study presents a cost comparison of Wilate® prophylaxis versus OD treatment in patients with Type 3 VWD and a severe bleeding phenotype, using real-world prospective data from the WIL-29 and WIL-31 phase 3 studies. A unique feature of this analysis is the intrapatient comparison: the same individuals were treated with OD therapy in WIL-29 (6 months) and subsequently received prophylactic treatment in WIL-31 (12 months), enabling robust, patient-level comparative data. Methods A lifetime horizon cost model was developed from both a U.S. payer and societal perspective. Clinical inputs were derived directly from WIL-29 and WIL-31 studies, including observed Wilate® dosing, breakthrough bleeding rates, and treatment consumption. Healthcare resource utilization (HCRU) costs for minor and major bleeds were estimated from published literature. Societal costs were calculated using bleed duration data from the studies, to estimate productivity loss. Subgroup analyses were conducted for adults and adult women with Type 3 VWD. All costs were adjusted to 2025 U.S. dollars. Results In the overall Type 3 VWD population with a severe bleeding phenotype, prophylactic use of Wilate® was cost-saving, with an average annual cost of $293,851, compared to $304,240 for OD treatment, resulting in annual savings of $10,389 per patient. Even greater annual savings were observed among all adults ($52,720/year) and adult women ($124,737/year). Over a lifetime, prophylaxis was projected to yield cumulative savings of $298,504 for the overall population, $1,514,820 for adults, and $3,584,070 for adult women. Inclusion of productivity loss further increased the annual economic advantage of prophylaxis by an additional $43,002 per patient per year. These findings highlight the substantial clinical and economic burden of OD therapy and the value of long-term prophylactic treatment in this patient population. Conclusions This is the first cost comparison of prophylaxis versus OD treatment in Type 3 VWD based on prospective, intrapatient data, a design that enhances reliability by minimizing variability across treatment phases. The model's strength lies in its real-world clinical inputs, integration of societal costs considerations, and focus on patients with severe bleeding phenotypes. The results demonstrate that Wilate® prophylaxis delivers not only meaningful clinical benefits as demonstrated in the clinical trials, but also significant cost-savings, especially among adult women where the economic impact was most pronounced. These findings support the expanded use of VWF concentrate prophylaxis within comprehensive VWD management strategies. Future research incorporating broader direct/indirect costs, including those related to long-term health outcomes, e.g., joint deterioration, chronic anaemia, heavy menstrual bleeds, mental health and quality-adjusted life years (QALYs), may further strengthen the economic and therapeutic value of prophylactic treatment.

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