OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare intravascular hematologic disorder characterized by uncontrolled terminal complement activation, thrombosis and organ damage. The terminal complement C5 inhibitor, ravulizumab, was approved by the FDA in 2018 for the intravenous treatment of PNH. A subcutaneous (SC) ravulizumab formulation (ravulizumab SC; 480 mg) is currently in clinical development which aims to provide a once weekly, self-administered treatment dose, compared with recently approved pegcetacoplan (1,080 mg); a proximal complement C3 inhibitor, self-administered SC twice weekly–every 3 days. This study aimed to estimate the treatment-related burden, defined as duration of treatment, and associated lost productivity costs for patients with PNH treated with either ravulizumab SC or pegcetacoplan in the United States.

METHODS: A cost-consequences model compared the aggregate duration of treatment and lost productivity costs (treatment duration × baseline wage [$20/hour]) associated with treatment with ravulizumab SC or pegcetacoplan over 2 years. Treatment duration included medication preparation, treatment infusion and recovery time. One self-administration training clinic visit, including travel time, was also included. Upper and lower estimates of each input, informed by prescribing information and assumptions, were created to accommodate treatment variation in pegcetacoplan dosing.

RESULTS: In 100 hypothetical patients, duration of treatment ranged from 2,895–3,075 hours for ravulizumab SC and from 12,253–43,373 hours for pegcetacoplan. Associated lost productivity costs ranged from $57,900–61,500 for ravulizumab SC and from $245,067–867,467 for pegcetacoplan.

CONCLUSIONS: Compared with ravulizumab SC, patients with PNH treated with pegcetacoplan were estimated to experience from 4.2–14.1 times more treatment-related burden, resulting in higher lost productivity costs. These differences in treatment-related burden will affect patients, healthcare providers, payers, and society.