Cost-Utility Analysis for Haemate-P in the Treatment of Von Willebrand Disease

OBJECTIVES: Von Willebrand Disease (vWD) is a blood disease caused by a deficiency of von Willebrand Factor (vWF), a fundamental protein in blood coagulation. It is the most common congenital bleeding disorder, and it is estimated to have a prevalence of symptomatic disease between 0,01% and 0,1%. This research aims to compare the cost-utility of Haemate-P with respect to its alternatives in the treatment of vWD.

METHODS: A cost-utility model was developed to estimate the incremental cost-effectiveness of treatments for patients with vWD aged >=12 years in Spain. Haemate-P was compared with Fahndi and Wilate in various treatment strategies: long-term prophylaxis, intermittent prophylaxis, and on-demand treatment. The model was based on a Markov design with no joint surgery, joint surgery, and death as health states. The Spanish National Health System perspective was adopted, and a lifetime time horizon was used with a cycle length of 6 months. The effectiveness in each arm was measured by linking a utility value to each state and patient’s age and a disutility value to each major bleeding event simulated. Both costs and utilities were discounted at 3%.

RESULTS: Under all treatment strategies, Haemate-P presents better ICER results than its alternative products (Fanhdi and Wilate). In prophylaxis, Haemate-P compared to Fanhdi was 1,313,845€ less expensive, with 0.13 additional QALYs, while the comparison against Wilate resulted in savings of 2,233,940€ and 0.29 additional QALYs. In intermittent prophylaxis, Haemate-P vs Fahndi and Wilate resulted in savings of 1,001,510€ and 1,520,998€, respectively, and 0.03 and 0.07 additional QALYs. Finally, on demand, Haemate-P had the same effectiveness as Fahndi and Wilate, but reduced the costs by 696,857€ and 1,145,780€.

CONCLUSIONS: Haemate-P presents better ICER results alternative under all treatment strategies, especially when used as a long-term prophylaxis treatment.