Cost-Utility Analysis of Ravulizumab Compared with Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria
| Autor: | Lufei Tu, Ioannis Tomazos, Thomas O'Connell, Marric Buessing, Scott J. Johnson, Simu K. Thomas |
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| Rok vydání: | 2020 |
| Předmět: |
Adult
Pediatrics medicine.medical_specialty Blood transfusion medicine.medical_treatment Cost-Benefit Analysis Population Hemoglobinuria Paroxysmal Antibodies Monoclonal Humanized 03 medical and health sciences 0302 clinical medicine Quality of life medicine Humans 030212 general & internal medicine Dosing education health care economics and organizations Pharmacology education.field_of_study Cost–utility analysis business.industry 030503 health policy & services Health Policy Public Health Environmental and Occupational Health Eculizumab medicine.disease Paroxysmal nocturnal hemoglobinuria Quality of Life Hemoglobinuria 0305 other medical science business medicine.drug |
| Zdroj: | PharmacoEconomics. 38(9) |
| ISSN: | 1179-2027 |
| Popis: | Paroxysmal nocturnal hemoglobinuria, characterized by intravascular hemolysis and venous thrombosis, can be managed with eculizumab, an inhibitor of the complement system; however, patients may periodically experience breakthrough hemolysis. Ravulizumab is a newly approved treatment for paroxysmal nocturnal hemoglobinuria that may reduce breakthrough hemolysis risk, thus improving health-related quality of life and reducing treatment costs. The objective of this study was to compare the costs and benefit of treatment with ravulizumab vs eculizumab in adult patients with paroxysmal nocturnal hemoglobinuria, from a US payer perspective. A cost-utility analysis was conducted using a semi-Markov model, informed by clinical experts. Lifetime costs and benefit (quality-adjusted life-years) (both discounted at 3% per annum) and incremental cost-effectiveness ratios were estimated, over a lifetime horizon. Results are reported for an entire treated population and subgroups of eculizumab treatment history. Scenario analyses were characterized by assumptions of non-inferiority between treatments, in terms of breakthrough hemolysis incidence and blood transfusion requirements, and of variations in eculizumab dosing adjustments used in response to breakthrough hemolysis. In the base-case analysis for the overall population, there was a positive impact on health-related quality of life (quality-adjusted life-year gain of 1.67) and costs were lower (− $1,673,465), for ravulizumab vs eculizumab. This led to a negative incremental cost-effectiveness ratio (− $1,000,818, indicating cost savings per quality-adjusted life-year gained). Health-related quality-of-life improvement and cost savings were also observed in all cohorts and scenario analyses. In adults with paroxysmal nocturnal hemoglobinuria, ravulizumab is associated with improved health-related quality of life and provides a large cost saving from the perspective of a US payer, when compared with eculizumab. |
| Databáze: | OpenAIRE |
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