Supplementary materials: The cost–effectiveness of pegcetacoplan in complement treatment-na¨ıve adults with paroxysmal nocturnal hemoglobinuria in the USA

These are peer-reviewed supplementary materials for the article 'The cost–effectiveness of pegcetacoplan in complement treatment-naive adults with paroxysmal nocturnal hemoglobinuria in the USA' published in the Journal of Comparative Effectiveness Research.Supplemental Table 1: Odds ratios between transfusion-avoidant and transfusion-required populations hemoglobin stabilization, pooled armsSupplemental Table 2: Probabilities for transfusion avoidant group – hemoglobin stabilizationSupplemental Table 3: Transition probabilities, comparators – hemoglobin stabilizationSupplemental Figure 1: Transfusion Required health state structureSupplemental Figure 2: Methodology of calculating transition probabilities for pegcetacoplan+BSC for (A) base-case model structure based on Hb normalization and (B) alternative model structure based on Hb stabilizationSupplemental Figure 3: Tornado diagrams of deterministic sensitivity analysis for pegcetacoplan versus (A) eculizumab and (B) ravulizumabSupplemental Figure 4: Incremental cost-effectiveness planes: (A) pegcetacoplan versus eculizumab, (B) pegcetacoplan versus ravulizumab – based on hemoglobin stabilizationSupplemental Figure 5: Distribution of patients over time simulated by the CEM for pegcetacoplan, eculizumab, and ravulizumabAim: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by hemolytic anemia, bone marrow failure and thrombosis, and is associated with high healthcare burden. We evaluated the cost–effectiveness of pegcetacoplan, a proximal complement-3 inhibitor (C3i), compared with the C5i, eculizumab and ravulizumab, in complement treatment-naive adults with PNH, from the US healthcare payer perspective. Materials & methods: A de novo cost–effectiveness model based on a Markov cohort structure evaluated lifetime (55-year) PNH costs and outcomes. The 6-month cycles of the model reflected the follow-up period of PRINCE (NCT04085601), an open-label trial of pegcetacoplan compared with eculizumab in C5i-naive patients. Data from PRINCE informed the clinical, safety and health-related quality of life outcomes in the model. Results: Pegcetacoplan was associated with lifetime cost savings of $1,176,808 and $213,062 relative to eculizumab and ravulizumab, respectively (largely attributed to reduced drug costs and blood transfusions), and additional quality-adjusted life years (QALYs) of 0.25 and 0.24. Conclusion: In patients with PNH who are treatment-naive, the base-case cost–effectiveness analysis, scenario analysis and sensitivity analysis showed both lifetime cost savings and increased QALYs associated with pegcetacoplan compared with eculizumab or ravulizumab in the USA.