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

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.Supplementary Table 1: Estimated utilities based on the Longworth and McKenzie algorithms Supplementary Table 2: Key scenario analysesSupplementary Table 3: Probability of developing adverse events per cycle Supplementary Table 4: Iron overload disutilitySupplementary Table 5: Number of physician visits/tests per cycle1 and unit healthcare costsSupplementary Table 6: Ravulizumab iron chelation costsSupplementary Table 7: Intravascular haemolysis breakthrough cost for patients who discontinue pegcetacoplan due to intravascular haemolysis breakthroughSupplementary Table 8: Unit costs of managing adverse eventsSupplementary Table 9: OWSA results for the 10 parameters that contribute the largest difference to the NMB for pegcetacoplan versus ravulizumabAim: 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

本数据集为发表于《比较有效性研究杂志》的论文《在英国环境下，与ravulizumab相比，pegcetacoplan治疗阵发性睡眠性血红蛋白尿症的成本效益》的同行评审补充材料。补充表1：基于Longworth和McKenzie算法的效用估计；补充表2：关键情景分析；补充表3：每周期发生不良事件的概率；补充表4：铁过载的不适效用；补充表5：每周期医师访问/测试次数及单位医疗成本；补充表6：ravulizumab铁螯合剂成本；补充表7：因血管内溶血突破而停用pegcetacoplan的患者血管内溶血突破成本；补充表8：管理不良事件的单位成本；补充表9：对pegcetacoplan与ravulizumab的NMB贡献最大的10个参数的OWSA结果。研究目的：阵发性睡眠性血红蛋白尿症（PNH）是一种罕见的血液疾病，以溶血性贫血、骨髓衰竭和血栓形成为特征。本研究评估了新型近端C3抑制剂pegcetacoplan与ravulizumab在PNH患者血红蛋白水平方面的成本效益。