Megias-Vericat JE, Escolar G, Wilson MR, Mendez P, McDade CL, Barrientos LV, Tomic R, Panebianco M, Linden S, Yan S. Cost-effectiveness and cost-utility analysis of haemate-P versus other von willebrand disease treatments in Spain. J Med Econ. 2025 Mar 7;28(1):436-45. doi: 10.1080/13696998.2025.2474886

BACKGROUND: 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 of -€1,313,845; incremental quality-adjusted life-years [QALY] of 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.