The cost-effectiveness of pegcetacoplan in complement treatment-naive adults with paroxysmal nocturnal hemoglobinuria in the USA

Aim: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by hemolytic anemia, bone marrow failure and thrombosis, and is associated with high healthcare burden. We evaluated the cost-effectiveness of pegcetacoplan, a proximal complement-3 inhibitor (C3i), compared with the C5i, eculizumab and ravulizumab, in complement treatment-naive adults with PNH, from the US healthcare payer perspective. Materials & methods: A de novo cost-effectiveness model based on a Markov cohort structure evaluated lifetime (55-year) PNH costs and outcomes. The 6-month cycles of the model reflected the follow-up period of PRINCE (NCT04085601), an open-label trial of pegcetacoplan compared with eculizumab in C5i-naive patients. Data from PRINCE informed the clinical, safety and health-related quality of life outcomes in the model. Results: Pegcetacoplan was associated with lifetime cost savings of $1,176,808 and $213,062 relative to eculizumab and ravulizumab, respectively (largely attributed to reduced drug costs and blood transfusions), and additional quality-adjusted life years (QALYs) of 0.25 and 0.24. Conclusion: In patients with PNH who are treatment-naive, the base-case cost- effectiveness analysis, scenario analysis and sensitivity analysis showed both lifetime cost savings and increased QALYs associated with pegcetacoplan compared with eculizumab or ravulizumab in the USA. Plain language summary What is this article about? Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease that can lead to a high level of illness in individuals who are affected with the genetic disorder. In the USA, patients who have PNH and severe symptoms or complications can currently receive one of three licensed treatments: pegcetacoplan, ravulizumab or eculizumab. However, in order to improve healthcare for these patients, some important information is needed to understand how well these treatments work and the total costs to the person or health insurer paying for the treatment. Therefore, this study measured the cost-effectiveness of the currently-available treatments for PNH. How was the study carried out? Using information from clinical trials and other published sources, this study looked at whether patients could benefit from improvements in life-expectancy and health-related quality of life of pegcetacoplan compared with the other treatments. These benefits were weighed against the costs of each of the drugs and other various healthcare costs. What do the results mean? The findings of this study suggest that pegcetacoplan is a treatment for PNH that is associated with more patient benefits and lower healthcare costs compared with ravulizumab and eculizumab.