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Cost-effectiveness and cost-utility analysis of Haemate-p versus other von Willebrand disease treatments in Spain
Megias-Vericat, J. E., Escolar, G., Wilson, M. R., Mendez, P., McDade, C. L., Barrientos, L. V., Tomic, R., Panebianco, M., Linden, S., & Yan, S. (2025). Cost-effectiveness and cost-utility analysis of Haemate-p versus other von Willebrand disease treatments in Spain. Journal of Medical Economics, 28(1), 436-445. Advance online publication. https://doi.org/10.1080/13696998.2025.2474886
OBJECTIVE: von Willebrand Disease (vWD) is the most common congenital bleeding disorder, with an estimated prevalence in Spain of 0.01%. The aim was to assess the cost-utility of Haemate-P compared with present alternatives in the treatment of vWD in Spain.
METHODS: A Markov model was developed in Microsoft Excel to estimate the cost-effectiveness of various treatments for vWD over a lifetime horizon. Transition probabilities among health states were based on age and number of bleeding events. Treatment strategies compared included Haemate-P, Fanhdi, and Wilate in long-term prophylaxis (LTP) or on-demand treatment (ODT). Costs and quality-of-life were measured based on patient age, treatment, and number of bleeding events incurred. Both costs and utilities were discounted at 3%. One-way and probabilistic sensitivity analyses were performed.
RESULTS: When comparing LTP regimens, Haemate-P was less costly and numerically more effective than both Fanhdi (incremental costs = -€1,313,845; incremental quality-adjusted life-years [QALY] = 0.13) and Wilate (incremental costs = -€2,233,940; incremental QALY = 0.29). For ODT, Haemate-P was assumed to have equal effectiveness as Fanhdi and Wilate but reduced the costs by €696,857 and €1,145,780, respectively. Haemate-P prophylaxis was more effective and less costly compared with Haemate-P on-demand in the base case (incremental costs = -€633,317; incremental QALY = 0.90). Results were generally robust to sensitivity analyses.
CONCLUSIONS: In patients with severe vWD experiencing a high bleed rate, Haemate-P prophylaxis is a less costly and potentially more effective treatment strategy and Haemate-P is cost-saving among on-demand strategies.