Cost-Effectiveness of Pegcetacoplan for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Patients With Treatment Failure After C5 Inhibitor Therapy: An Analysis From the Brazilian Public Healthcare System Perspective
Author(s)
Mauricio Penaquio, MBA1, Nilson Carvalho, MBA1, Valnei Canutti, Dr.1, Gabriel Leonel Marasco, MBA2, Camila Pepe, MSc2, Fernanda Bertasi, MBA1.
1Pint Pharma brazil, São Paulo, Brazil, 2Origin Health, São Paulo, Brazil.
1Pint Pharma brazil, São Paulo, Brazil, 2Origin Health, São Paulo, Brazil.
OBJECTIVES: To evaluate the cost-effectiveness of pegcetacoplan compared with eculizumab and ravulizumab for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who remain anemic despite prior use of C5 inhibitors from the perspective of the Brazilian public healthcare system (SUS).
METHODS: A cost-utility analysis was conducted using a Markov model with monthly cycles and a lifetime horizon to simulate the clinical and economic outcomes of pegcetacoplan compared with eculizumab and ravulizumab, the two C5 inhibitors currently available in SUS. The model included five mutually exclusive health states based on hemoglobin levels and transfusion requirements: Hb ≥12 g/dL, Hb 10-12 g/dL, Hb <10 g/dL without transfusion, Hb <10 g/dL with transfusion, and death. Transition probabilities were derived from the PEGASUS randomized controlled trial (pegcetacoplan vs. eculizumab) and a matching-adjusted indirect comparison (pegcetacoplan vs. ravulizumab). Utilities were sourced from published literature. Costs included drug acquisition, administration, monitoring, transfusions, and complication management. The analysis considered a 5% annual discount rate for both costs and outcomes. Deterministic and probabilistic sensitivity analyses were performed to assess uncertainty.
RESULTS: Pegcetacoplan was associated with an increase of 1.04 QALYs and 0.89 life years compared with eculizumab and an increase of 1.01 QALYs and 0.87 life years compared with ravulizumab. Total costs for pegcetacoplan were 27.63% and 13.46% lower when compared to eculizumab and ravulizumab, respectively, over the lifetime horizon, driven primarily by reduced need for transfusions and lower overall treatment and management costs. As a result, pegcetacoplan was found to be a dominant strategy. Probabilistic sensitivity analyses confirmed the robustness of these findings.
CONCLUSIONS: Pegcetacoplan demonstrated superior effectiveness and cost savings relative to eculizumab and ravulizumab in the treatment of PNH patients with persistent anemia after C5 inhibitor therapy. These results indicate that pegcetacoplan is a cost-effective and potentially cost-saving alternative in the Brazilian public healthcare system.
METHODS: A cost-utility analysis was conducted using a Markov model with monthly cycles and a lifetime horizon to simulate the clinical and economic outcomes of pegcetacoplan compared with eculizumab and ravulizumab, the two C5 inhibitors currently available in SUS. The model included five mutually exclusive health states based on hemoglobin levels and transfusion requirements: Hb ≥12 g/dL, Hb 10-12 g/dL, Hb <10 g/dL without transfusion, Hb <10 g/dL with transfusion, and death. Transition probabilities were derived from the PEGASUS randomized controlled trial (pegcetacoplan vs. eculizumab) and a matching-adjusted indirect comparison (pegcetacoplan vs. ravulizumab). Utilities were sourced from published literature. Costs included drug acquisition, administration, monitoring, transfusions, and complication management. The analysis considered a 5% annual discount rate for both costs and outcomes. Deterministic and probabilistic sensitivity analyses were performed to assess uncertainty.
RESULTS: Pegcetacoplan was associated with an increase of 1.04 QALYs and 0.89 life years compared with eculizumab and an increase of 1.01 QALYs and 0.87 life years compared with ravulizumab. Total costs for pegcetacoplan were 27.63% and 13.46% lower when compared to eculizumab and ravulizumab, respectively, over the lifetime horizon, driven primarily by reduced need for transfusions and lower overall treatment and management costs. As a result, pegcetacoplan was found to be a dominant strategy. Probabilistic sensitivity analyses confirmed the robustness of these findings.
CONCLUSIONS: Pegcetacoplan demonstrated superior effectiveness and cost savings relative to eculizumab and ravulizumab in the treatment of PNH patients with persistent anemia after C5 inhibitor therapy. These results indicate that pegcetacoplan is a cost-effective and potentially cost-saving alternative in the Brazilian public healthcare system.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE265
Topic
Economic Evaluation
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)