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Thank you for attending THSNA 2026. The virtual meeting is now closed.
Presentation Details
| Comparative Clinical and Economic Evaluation of On-Demand versus Prophylactic Treatment with pdVWF/FVIII 1:1 and Recombinant VWF in Adults with Type 3 von Willebrand Disease in the United States Robert F Sidonio JR1, Nathan T Connell2, 3, 4, Fernando Corrales-Medina5, Caroline Malcolmson6. 1Emory University School of Medicine, Atlanta, GA, USA.2Boston Bleeding Disorders Center, Boston, MA, USA.3Brigham and Women's Hospital, Boston, MA, USA.4Harvard Medical School, Boston, MA, USA.5University of Miami-Miller School of Medicine, Miami, FL, USA.6University of Toronto, Toronto, ON, USA |
Abstract
Background: Patients with type 3 von Willebrand disease (VWD) experience severe, recurrent bleeding requiring long-term replacement therapy with von Willebrand factor (VWF) concentrates. In the United States, plasma-derived VWF/FVIII in a 1:1 activity ratio (pdVWF/FVIII 1:1; wilate®) and recombinant VWF (rVWF; Vonvendi®) are the only concentrates approved for prophylaxis. While prophylaxis substantially reduces annualized bleeding rates (ABR) compared with on-demand (OD) treatment, data comparing their relative cost-effectiveness remain limited. This analysis integrates intrapatient evidence to assess both the clinical impact and economic value of prophylaxis versus OD therapy and to compare costs between wilate and rVWF prophylaxis. Objectives: To evaluate (1) the clinical and economic outcomes of wilate prophylaxis versus OD therapy in adults with severe type 3 VWD, and (2) the comparative prophylaxis costs of wilate versus rVWF from a U.S. payer perspective. Methods: A lifetime-horizon cost model was developed using clinical data from the WIL-29 (OD phase) and WIL-31 (prophylaxis phase) studies, in which the same patients received both regimens sequentially. Annual dosing and breakthrough bleeding data for wilate and rVWF were taken from pivotal trials (WIL-31 and NCT02973087, respectively), and all cost calculations were performed assuming a fictional 70-kg patient. Costs were adjusted to 2025 U.S. dollars using Medi-Span average sales prices (ASPs): $1.271 per IU for wilate and $1.846 per IU for rVWF. Health-care resource utilization for minor/major bleeds and productivity loss estimates were derived from published U.S. sources. Analyses were conducted from both payer and societal perspectives. Results: Prophylaxis (WIL-31) vs On-Demand (WIL-29): In adults with severe type 3 VWD, wilate prophylaxis reduced median total ABR by 93.6% (from 29.7 to 1.9 events/year) and decreased annual treatment costs from $381,243 (OD) to $328,522 (prophylaxis), yielding $52,720 in yearly savings per patient. When societal costs (e.g., work loss) were included, the annual economic advantage of prophylaxis increased by an additional $44,112 per patient. Among adult type 3 females, prophylaxis generated $124,737 in annual savings from the payer perspective, which increased by $22,843 when societal costs were included. wilate vs rVWF Prophylaxis: Mean weekly wilate dosing (61.8 IU/kg) resulted in an annual prophylaxis cost of $285,960 ± $90,216, compared with $622,000 ± $227,789 for rVWF (92.6 IU/kg), representing a 54% lower cost for wilate. Even assuming identical dosing, wilate remained 31% less costly ($357,000 vs $519,000). Breakthrough-bleed management costs were modest ($7,880 wilate vs $13,970 rVWF). Overall, combining OD-to-prophylaxis transition data and product-level comparisons demonstrates that prophylaxis markedly improves bleeding control and delivers substantial cost savings across clinical scenarios. Conclusion: wilate prophylaxis provides robust clinical benefits and significant cost advantages compared with OD therapy and rVWF prophylaxis in adults with severe type 3 VWD. These findings, supported by prospective intrapatient data and current U.S. pricing, indicate that wilate is a cost-efficient, first-line prophylactic option from both payer and societal perspectives in the US. Future analyses incorporating quality-adjusted life years and indirect health outcomes (e.g., joint preservation, chronic anemia prevention) will further define the long-term economic and therapeutic value of prophylaxis in this population.
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No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.