ECONOMIC EVALUATION OF PEGCETACOPLAN FOR PNH FROM THE BRAZILIAN PUBLIC HEALTHCARE SYSTEM PERSPECTIVE
Author(s)
Fernanda Bertasi, MBA1, Nilson Carvalho, MBA1, Marina Fusco, MBA1, Mauricio Penaquio, MBA1, Amer Omar, MBA2, Liliane Victoria, MBA3, Erika Camargo, MBA3, Gabriel L. Marasco, MBA4, Camila L. Pepe, MSc4;
1Pint Pharma Brazil, São Paulo, Brazil, 2Swedish orphan Biovitrium AG, Basel, Switzerland, 3Choices, São Paulo, Brazil, 4Origin Health, São Paulo, Brazil
1Pint Pharma Brazil, São Paulo, Brazil, 2Swedish orphan Biovitrium AG, Basel, Switzerland, 3Choices, São Paulo, Brazil, 4Origin Health, São Paulo, Brazil
OBJECTIVES: Background: Paroxysmal nocturnal hemoglobinuria (PNH) is associated with high clinical and economic burden due to chronic hemolysis, transfusion dependence, complications, and long-term treatment costs. Current therapies in Brazil rely on complement C5 inhibitors, which present substantial expenditure and may require continued transfusion support. Objectives: To evaluate the cost-effectiveness and budgetary impact of pegcetacoplan for adult PNH patients previously treated with complement inhibitors, from the perspective of the Brazilian Public Healthcare System.
METHODS: Methods: A cost-utility analysis was developed using a lifetime horizon with 4-week cycles and a 5% annual discount rate for costs and outcomes. Comparators included eculizumab, ravulizumab, and iptacopan. Health states incorporated transfusion status, hemoglobin levels, and death. Costs considered drug acquisition, administration, complications, transfusion, monitoring, diagnostics, and vaccination. Sensitivity analyses were conducted to test model robustness. A 5-year budget impact analysis estimated eligible population, market share progression, and treatment costs for pegcetacoplan versus current C5 inhibitor utilization.
RESULTS: Results: Pegcetacoplan was dominant versus eculizumab and ravulizumab, demonstrating lower lifetime costs combined with greater effectiveness in quality-adjusted life years. When compared with iptacopan, pegcetacoplan resulted in similar effectiveness with lower total cost. Drug acquisition price and utility values generated the highest influence in sensitivity analyses, yet the dominance findings remained consistent. Budget impact analysis showed that implementation of pegcetacoplan could reduce public expenditure: average annual savings of approximately R$16 million with an initial 30% market share increasing to 58% over five years, and up to R$27 million annually in an alternative scenario with 50-70% uptake.
CONCLUSIONS: Conclusion: From the Brazilian Public Healthcare System perspective, pegcetacoplan represents a cost-saving therapeutic strategy for PNH, yielding lower long-term treatment costs than available C5 inhibitors and offering meaningful reduction in projected national expenditures. These findings support pegcetacoplan as an economically advantageous option for managing previously treated PNH patients.
METHODS: Methods: A cost-utility analysis was developed using a lifetime horizon with 4-week cycles and a 5% annual discount rate for costs and outcomes. Comparators included eculizumab, ravulizumab, and iptacopan. Health states incorporated transfusion status, hemoglobin levels, and death. Costs considered drug acquisition, administration, complications, transfusion, monitoring, diagnostics, and vaccination. Sensitivity analyses were conducted to test model robustness. A 5-year budget impact analysis estimated eligible population, market share progression, and treatment costs for pegcetacoplan versus current C5 inhibitor utilization.
RESULTS: Results: Pegcetacoplan was dominant versus eculizumab and ravulizumab, demonstrating lower lifetime costs combined with greater effectiveness in quality-adjusted life years. When compared with iptacopan, pegcetacoplan resulted in similar effectiveness with lower total cost. Drug acquisition price and utility values generated the highest influence in sensitivity analyses, yet the dominance findings remained consistent. Budget impact analysis showed that implementation of pegcetacoplan could reduce public expenditure: average annual savings of approximately R$16 million with an initial 30% market share increasing to 58% over five years, and up to R$27 million annually in an alternative scenario with 50-70% uptake.
CONCLUSIONS: Conclusion: From the Brazilian Public Healthcare System perspective, pegcetacoplan represents a cost-saving therapeutic strategy for PNH, yielding lower long-term treatment costs than available C5 inhibitors and offering meaningful reduction in projected national expenditures. These findings support pegcetacoplan as an economically advantageous option for managing previously treated PNH patients.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE167
Topic
Economic Evaluation
Topic Subcategory
Budget Impact Analysis
Disease
SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)