Supplementary tables: The cost–effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting

https://doi.org/10.6084/m9.figshare.25746585

dataset

posted on 2024-05-03, 14:16 authored by Zalmai Hakimi, Koo Wilson, Eoin McAughey, Michal Pochopien, Piotr Wojciechowski, Mondher Toumi, Chris Knight, Sujata Sarda, Nikita Patel, Catherine Wiseman, Nuno Pinto de Castro, Jameel Nazir, Richard Kelly

These are peer-reviewed supplementary materials for the article 'The cost–effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting' published in the Journal of Comparative Effectiveness Research.<ul><li>Supplementary Table 1: Estimated utilities based on the Longworth and McKenzie algorithms </li><li>Supplementary Table 2: Key scenario analyses</li><li>Supplementary Table 3: Probability of developing adverse events per cycle </li><li>Supplementary Table 4: Iron overload disutility</li><li>Supplementary Table 5: Number of physician visits/tests per cycle1 and unit healthcare costs</li><li>Supplementary Table 6: Ravulizumab iron chelation costs</li><li>Supplementary Table 7: Intravascular haemolysis breakthrough cost for patients who discontinue pegcetacoplan due to intravascular haemolysis breakthrough</li><li>Supplementary Table 8: Unit costs of managing adverse events</li><li>Supplementary Table 9: OWSA results for the 10 parameters that contribute the largest difference to the NMB for pegcetacoplan versus ravulizumab</li></ul>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.