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 analyses
- Supplementary Table 3: Probability of developing adverse events per cycle
- Supplementary Table 4: Iron overload disutility
- Supplementary Table 5: Number of physician visits/tests per cycle1 and unit healthcare costs
- Supplementary Table 6: Ravulizumab iron chelation costs
- Supplementary Table 7: Intravascular haemolysis breakthrough cost for patients who discontinue pegcetacoplan due to intravascular haemolysis breakthrough
- Supplementary Table 8: Unit costs of managing adverse events
- Supplementary Table 9: OWSA results for the 10 parameters that contribute the largest difference to the NMB for pegcetacoplan versus ravulizumab
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.
