Cost-effectiveness and cost-utility analysis of Haemate-P versus other von Willebrand disease treatments in Spain

Autores de IIS La Fe

• Juan Eduardo Megías Vericat

  Autor

Participantes ajenos a IIS La Fe

• Escolar G.
• Wilson M.R.
• Mendez P.
• McDade C.L.
• Barrientos L.V.
• Tomic R.
• Panebianco M.
• Linden S.
• Yan S.

Grupos

• Grupo de Investigación de Farmacia

Abstract

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. © 2025 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.

Keywords

• Adolescent; Adult; Cost-Benefit Analysis; Drug Combinations; Factor VIII; Female; Hemorrhage; Humans; Male; Markov Chains; Middle Aged; Models, Econometric; Quality of Life; Quality-Adjusted Life Years; Spain; von Willebrand Diseases; von Willebrand Factor; blood clotting factor 8 plus von Willebrand factor; blood clotting factor 8; factor VIII, von Willebrand factor drug combination; von Willebrand factor; adolescent; adult; aged; Article; bleeding prophylaxis; child; comparative study; controlled study; cost; cost effectiveness analysis; cost utility analysis; drug cost; economic model; female; groups by age; health status; hemarthrosis; human; infant; joint surgery; major bleeding; major clinical study; male; Markov chain; middle aged; minor bleeding; probability; quality adjusted life year; quality of life; sensitivity analysis; Spain; treatment duration; very elderly; von Willebrand disease; bleeding; cost benefit analysis; drug combination; drug therapy; econometric model; econom