OBJECTIVES

Eculizumab and ravulizumab are the only approved treatments that prevent intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH). Ravulizumab was engineered from eculizumab to extend the duration of complete complement C5 inhibition, thereby increasing the dosing interval and reducing the risk of breakthrough hemolysis due to incomplete free C5 suppression. We developed a cost-utility model of ravulizumab versus eculizumab in adult patients with PNH.

METHODS

The model structure was informed by literature review, expert clinical input and clinical trial data. PNH-related outcomes modeled over a lifetime included: current, historical or no breakthrough hemolysis (related to incomplete free C5 suppression, infection-related, or undetermined), eculizumab dosage, remission, and avoidance of blood transfusion. Differences in clinical outcomes between treatments were modeled based on two randomized, 26-week, phase 3 studies of ravulizumab versus eculizumab. Costs were considered from the healthcare-system perspective and benefits were considered in terms of patient health-related quality of life (estimated via mapping the European Organization for Research and Treatment of Cancer QLQ-C30 to the EQ-5D). The modeled population comprised two cohorts: 1, eculizumab-naïve; 2, stable on eculizumab at labelled dosage. Results presented are the mean across both cohorts.

RESULTS

In the base case, mean incremental quality-adjusted life years (QALYs) for ravulizumab versus eculizumab were 1.06 and mean incremental costs were USD (2018) –1 673 465, reflecting economic ‘dominance’ of ravulizumab (higher QALYs, lower costs). Ravulizumab was dominant in 93.7% of Monte-Carlo simulations. In univariate sensitivity analyses, results were most responsive to eculizumab dosage and health-utility variation between treatments.

CONCLUSIONS

This model demonstrated that ravulizumab is cost-saving compared with eculizumab for the management of adult patients with PNH, at US list prices. In addition, ravulizumab was predicted to decrease the burden of treatment and improve overall quality of life compared with eculizumab in adult patients with PNH.