The cost-effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting

Zalmai Hakimi, Koo Wilson, Eoin McAughey, Michal Pochopien, Piotr Wojciechowski, Mondher Toumi, Chris Knight, Sujata P Sarda, Nikita Patel, Catherine Wiseman, Nuno Pinto de Castro, Jameel Nazir, Richard J Kelly, Zalmai Hakimi, Koo Wilson, Eoin McAughey, Michal Pochopien, Piotr Wojciechowski, Mondher Toumi, Chris Knight, Sujata P Sarda, Nikita Patel, Catherine Wiseman, Nuno Pinto de Castro, Jameel Nazir, Richard J Kelly

Abstract

Aim: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by hemolytic anemia, bone marrow failure and thrombosis. We evaluated, the cost-effectiveness of pegcetacoplan, a novel proximal C3 inhibitor, versus ravulizumab in patients with PNH and hemoglobin levels <10.5 g/dl despite eculizumab treatment in the UK healthcare and social services setting. Materials & methods: A Markov cohort framework model, based on the data from the pivotal trial of pegcetacoplan (PEGASUS/NCT03500549), evaluated lifetime costs and outcomes. Patients transitioned through 3 PNH hemoglobin level/red blood cell transfusion health states. Results: Pegcetacoplan provides lower lifetime costs/greater quality-adjusted life years (£6,409,166/14.694QALYs, respectively) versus ravulizumab (£6,660,676/12.942QALYs). Conclusion: Pegcetacoplan is associated with enhanced anemia control, greater QALYs and reduced healthcare costs versus ravulizumab in the UK healthcare and social services setting.

Keywords: cost–effectiveness; hemolytic anemia; paroxysmal nocturnal hemoglobinuria; pegcetacoplan; red blood cell transfusion.

Source: PubMed

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