Cost‐effectiveness of recombinant factor VIII Fc versus emicizumab for prophylaxis in adults and adolescents with haemophilia A without inhibitors in the UK

• Published in: European journal of haematology 2023-03, Vol.110 (3), p.262-270
• Main Authors: Kragh, Nana, Tytula, Anna, Pochopien, Michał, Aballéa, Samuel, Toumi, Mondher, Hakimi, Zalmai, Nazir, Jameel, Bystrická, Linda, Fatoye, Francis
• Format: Article
• Language: English
• Subjects: Adolescent; Adolescents; Adult; Coagulation factors; Cost-Benefit Analysis; cost‐effectiveness analysis; Disease prevention; emicizumab; Factor VIII - therapeutic use; Fc receptors; Fusion protein; haemophilia A; Hemophilia; Hemophilia A - therapy; Humans; Male; Original; Patients; Prophylaxis; Prostate-Specific Antigen - therapeutic use; recombinant factor VIII Fc; Recombinant Fusion Proteins - therapeutic use; Sensitivity analysis; United Kingdom
• ISSN: 0902-4441; 1600-0609
• DOI: 10.1111/ejh.13901

Introduction The economic and clinical burden of haemophilia A is high. Primary prophylaxis with factor VIII replacement therapy is the recognised standard of care, but the emergence of non‐factor therapies, such as emicizumab, is extending treatment options for people with haemophilia A. Aim There are currently no direct comparisons of efficacy or cost between recombinant factor FVIII Fc‐fusion protein efmoroctocog alfa (a recombinant factor FVIII Fc‐fusion protein referred to herein as rFVIIIFc) and emicizumab; therefore, a cost‐effectiveness model was developed to compare prophylactic treatment with rFVIIIFc versus emicizumab in patients with haemophilia A without inhibitors in the UK. Methods The cost‐effectiveness model was based on a matching‐adjusted indirect comparison and included male patients, aged ≥12 years, with haemophilia A without inhibitors. The model was designed as a Markov process with a flexible lifelong time horizon, and cost‐effectiveness was presented as an incremental cost‐effectiveness ratio. Base‐case analysis and sensitivity analyses (including scenario analyses, one‐way deterministic sensitivity analysis [DSA] and probability sensitivity analysis [PSA]) were performed using the following treatment strategies: individualised prophylaxis with rFVIIIFc and prophylaxis with emicizumab administered once weekly (scenario analyses used regimens of once every 2 weeks or once every 4 weeks). Results Base‐case analysis, DSA and PSA indicated that, compared with emicizumab administered once weekly, rFVIIIFc individualised prophylaxis was the dominant treatment strategy, with lower costs, a greater number of quality‐adjusted life years, and a lower number of bleeds. Conclusions rFVIIIFc has proven efficacy and is cost‐effective compared with emicizumab, providing clinicians with a viable treatment option to improve the health outcomes for adults and adolescents with haemophilia A in the UK.