Open AccessThe cost–effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting
Author(s) -
Zalmaï Hakimi,
Koo Wilson,
Eoin McAughey,
M. Pochopień,
Piotr Wojciechowski,
Mondher Toumi,
Chris Knight,
Sujata P. Sarda,
Nikita Patel,
Catherine Wiseman,
Nuno Pinto de Castro,
Jameel Nazir,
Richard Kelly
Publication year - 2022
Publication title -
journal of comparative effectiveness research
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.567
H-Index - 23
eISSN - 2042-6313
pISSN - 2042-6305
DOI - 10.2217/cer-2022-0076
Subject(s) - paroxysmal nocturnal hemoglobinuria , medicine , eculizumab , hemoglobinuria , hemoglobin , pediatrics , cohort , anemia , intensive care medicine , immunology , antibody , complement system
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.