Cost-Utility Analysis of Ravulizumab Compared with Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria

• Published in: PharmacoEconomics Vol. 38; no. 9; pp. 981 - 994
• Main Authors: O’Connell, Thomas, Buessing, Marric, Johnson, Scott, Tu, Lufei, Thomas, Simu K., Tomazos, Ioannis
• Format: Journal Article
• Language: English
• Published: Cham Springer International Publishing 01-09-2020; Springer; Springer Nature B.V
• Subjects: Analysis; Bone marrow; Clinical trials; Cost analysis; Cost control; Drug therapy; Economic aspects; Health Administration; Health Economics; Health technology assessment; Hemoglobinuria, Paroxysmal; Infections; Medical care, Cost of; Medicine; Medicine & Public Health; Monoclonal antibodies; Original Research Article; Patients; Pharmacoeconomics and Health Outcomes; Public Health; Quality of life; Quality of Life Research; Studies; Thrombosis; United States; United States > US
• ISSN: 1170-7690; 1179-2027
• DOI: 10.1007/s40273-020-00929-z

Background
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.
Objective
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.
Methods
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.
Results
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.
Conclusions
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.