Artikel ; Online: Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors.
2024 Band 41, Heft 6, Seite(n) 2307–2323
Abstract: 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 ... ...
Abstract | 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. |
---|---|
Mesh-Begriff(e) | Humans ; Hemophilia B/drug therapy ; Hemophilia B/economics ; Factor IX/therapeutic use ; Factor IX/economics ; Cost-Benefit Analysis ; Male ; Recombinant Fusion Proteins/economics ; Recombinant Fusion Proteins/therapeutic use ; Immunoglobulin Fc Fragments/therapeutic use ; Immunoglobulin Fc Fragments/economics ; Quality-Adjusted Life Years ; Adolescent ; Adult ; Markov Chains ; Hemorrhage/prevention & control ; Child ; Young Adult ; Recombinant Proteins/therapeutic use ; Recombinant Proteins/economics ; Italy ; Middle Aged |
Chemische Substanzen | Factor IX (9001-28-9) ; Recombinant Fusion Proteins ; Immunoglobulin Fc Fragments ; factor IX Fc fusion protein ; Recombinant Proteins |
Sprache | Englisch |
Erscheinungsdatum | 2024-04-23 |
Erscheinungsland | United States |
Dokumenttyp | Journal Article |
ZDB-ID | 632651-1 |
ISSN | 1865-8652 ; 0741-238X |
ISSN (online) | 1865-8652 |
ISSN | 0741-238X |
DOI | 10.1007/s12325-024-02841-w |
Datenquelle | MEDical Literature Analysis and Retrieval System OnLINE |
Volltext online
Zusatzmaterialien
Kategorien
Verfügbar in ZB MED Köln/Königswinter
Zs.A 2101: Hefte anzeigen | Standort: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (1.OG) ab Jg. 2022: Lesesaal (EG) |
Über subito bestellen
Dieser Service ist kostenpflichtig (siehe Lieferbedingungen von subito). Bestellungen, die einen Artikel nebst Supplementary Material umfassen, werden grundsätzlich wie mehrfache Bestellungen bearbeitet. Gebühren fallen in diesen Fällen für jede einzelne Bestellung an.