Cost Minimization for the Treatment of Hemophilia A
Keywords:
hemophilia A; cost minimization analysis; coagulation factor VIII; emicizumab.Abstract
Introduction: Hemophilia A is a rare disease caused by the absence or defect of coagulation factor VIII, which leads to coagulation and bleeding problems in patients suffering from the condition.
Objectives: To describe recent studies involving cost minimization analyses for Hemophilia A.
Methods: A literature review was conducted using PubMed and Google Scholar covering the period from 2019 to 2024. Keywords such as "hemophilia A," "cost minimization analysis," "coagulation factor FVIII," "emicizumab," and "pharmacoeconomics" were employed. Articles and conference abstracts published in English were included. Additionally, the selection of articles was supplemented with works describing the pathophysiology of Hemophilia A and the rationale behind the therapies available for its treatment.
Results: Replacement therapy with coagulation factor VIII is more cost-effective for treating patients with Hemophilia A, specifically those without inhibitors, compared to emicizumab. Furthermore, when comparing the various variants and formulations of coagulation factor VIII, a trend was observed indicating that products featuring a modified structure, specifically those with an extended half-life, are the most advantageous options.
Conclusions: Replacement therapy with coagulation factor VIII represents the most affordable option for patients without inhibitors. Coagulation factor VIII products with an extended half-life and structural modifications offer both clinical and economic advantages. These findings underscore the importance of considering both costs and efficacy when making therapeutic decisions regarding Hemophilia A.
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