Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors

• Published in: Advances in therapy 2024-06, Vol.41 (6), p.2307-2323
• Main Authors: Pochopien, Michal, Tytuła, Anna, Toumi, Mondher, Falk, Aletta, Martone, Nicoletta, Hakimi, Zalmai, Eriksson, Daniel
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Cardiology; Child; Cost-Benefit Analysis; Endocrinology; Factor IX - economics; Factor IX - therapeutic use; Hemophilia B - drug therapy; Hemophilia B - economics; Hemorrhage - prevention & control; Humans; Immunoglobulin Fc Fragments - economics; Immunoglobulin Fc Fragments - therapeutic use; Internal Medicine; Italy; Male; Markov Chains; Medicine; Medicine & Public Health; Middle Aged; Oncology; Original Research; Pharmacology/Toxicology; Quality-Adjusted Life Years; Recombinant Fusion Proteins - economics; Recombinant Fusion Proteins - therapeutic use; Recombinant Proteins - economics; Recombinant Proteins - therapeutic use; Rheumatology; Young Adult
• ISSN: 0741-238X; 1865-8652; 1865-8652
• DOI: 10.1007/s12325-024-02841-w

Introduction Recombinant factor IX (rFIX) and recombinant FIX Fc fusion protein (rFIXFc) are standard half-life and extended half-life FIX replacement therapies, respectively, and represent established treatment options indicated for adults and children with haemophilia B. These FIX replacement therapies can be administered as prophylaxis (to prevent bleeding) or ‘on-demand’ (to stop bleeding). This analysis aimed to estimate the cost-effectiveness of once-weekly prophylaxis with rFIXFc versus on-demand treatment with rFIX in patients with haemophilia B without inhibitors in the Italian healthcare setting. Methods A Markov model was developed to assess a hypothetical cohort of adolescent or adult male patients (≥ 12 years) with haemophilia B (FIX level of ≤ 2 IU/dL) without inhibitors. Model inputs were derived from the pivotal phase 3 clinical studies for rFIXFc and rFIX, published literature and assumptions when published data were unavailable. The model employed a lifelong time horizon with 6-monthly transitions between health states, and it estimated total costs, total quality-adjusted life years (QALYs), number of bleeds, number of surgeries and incremental cost-effectiveness ratio. Results rFIXFc prophylaxis was associated with lower total costs per patient (€5,308,625 versus €6,564,510) and greater total QALYs per patient (15.936 versus 11.943) compared with rFIX on-demand; rFIXFc prophylaxis was therefore the dominant treatment strategy. The model also demonstrated that rFIXFc prophylaxis was associated with fewer incremental bleeds (− 682.29) and surgeries (− 0.39) compared with rFIX on-demand. Conclusions rFIXFc prophylaxis provides improved health outcomes and lower costs, and represents a cost-effective treatment option compared with rFIX on-demand for adolescent and adult male patients with haemophilia B. This comparative assessment of cost-effectiveness should help to inform both clinicians and healthcare policy makers when making treatment decisions for patients with haemophilia B.