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

<b>These are peer-reviewed supplementary materials for the article '</b><b>The cost–effectiveness, of pegcetacoplan </b><b>compared with ravulizumab for the </b><b>treatment of paroxysmal nocturnal </b><b>hemoglobinuria, in a UK setting</b><b>' published in the</b><b> </b><b><i>Journal of Comparative Effectiveness Research</i></b><b>.</b><b>Supplementary Table 1: </b>Estimated utilities based on the Longworth and McKenzie algorithms <b>Supplementary Table 2: </b>Key scenario analyses<b>Supplementary Table 3: </b>Probability of developing adverse events per cycle <b>Supplementary Table 4: </b>Iron overload disutility<b>Supplementary Table 5: </b>Number of physician visits/tests per cycle1 and unit healthcare costs<b>Supplementary Table 6: </b>Ravulizumab iron chelation costs<b>Supplementary Table 7: </b>Intravascular haemolysis breakthrough cost for patients who discontinue pegcetacoplan due to intravascular haemolysis breakthrough<b>Supplementary Table 8: </b>Unit costs of managing adverse events<b>Supplementary Table 9: </b>OWSA results for the 10 parameters that contribute the largest difference to the NMB for pegcetacoplan versus ravulizumab<b>Aim:</b> 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 &lt;10.5 g/dl despite eculizumab treatment in the UK healthcare and social services setting. <b>Materials &amp; methods: </b>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. <b>Results:</b> Pegcetacoplan provides lower lifetime costs/greater quality-adjusted life years (£6,409,166/14.694QALYs, respectively) versus ravulizumab (£6,660,676/12.942QALYs). <b>Conclusion:</b> Pegcetacoplan is associated with enhanced anemia control, greater QALYs and reduced healthcare costs versus ravulizumab in the UK healthcare and social services setting.