Abstract / Summary
Abstract: Von Willebrand factor (VWF) concentrate prophylaxis is recommended for patients with severe von Willebrand disease (VWD) or VWD with frequent bleeding symptoms but remains underused due, in part, to the high direct cost associated with regular concentrate administration. Robust cost-effectiveness data for VWF prophylaxis are limited, with most analyses relying on literature-based assumptions rather than prospective clinical data. A trial-based cost-effectiveness analysis was developed to estimate the long-term economic impact of plasma-derived VWF/factor VIII (Wilate) prophylaxis vs on-demand therapy in patients with type 3 VWD in the United States, from both payer and societal perspectives. Clinical inputs were derived directly from phase 3 clinical studies, whereas health care costs were obtained from published sources. Productivity losses during bleeding events were incorporated into the societal analysis. Subgroup analyses were performed for adults and adult women with type 3 VWD, and evaluations were conducted for both 1-year and lifetime horizons. The payer analysis highlighted potential cost savings with Wilate prophylaxis across all groups, with estimated annual savings of $10 898 per individual in the overall type 3 VWD cohort, $53 264 among adults, and $125 712 among women. Savings were higher in the societal perspective, with the largest benefit observed in women ($148 555 per individual annually). The primary cost drivers were bleeding rates and treatment costs during on-demand therapy. In addition to its established clinical efficacy, these findings demonstrate a potential substantial economic advantage of Wilate prophylaxis and support broader adoption of VWF-based prophylaxis for individuals with type 3 VWD in the United States.