Cost-Utility Analysis of Iptacopan for Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Previously Treated With C5 Inhibitor in the Brazilian Private Healthcare System
Author(s)
Diego Kashiura, BPharm1, Bruna Zanotto, BPharm, MSc2, Lucas Torres, BPharm, MSc1, Anggie Wiyani, BPharm, MSc3, Cheryl Druchok, BScKin, MASc, PhD4.
1Novartis, São Paulo, Brazil, 2HTAnalyze, Porto Alegre, Brazil, 3Novartis, London, United Kingdom, 4Eversana, Burlington, ON, Canada.
1Novartis, São Paulo, Brazil, 2HTAnalyze, Porto Alegre, Brazil, 3Novartis, London, United Kingdom, 4Eversana, Burlington, ON, Canada.
OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare disease characterized by chronic hemolytic anemia. In Brazil's private healthcare system, the standard of care is C5 inhibitors (C5i), with ravulizumab as the only option on the mandatory coverage list. Despite the advances in PNH care, limitations with persistent anemia and frequent treatment infusions remain. Iptacopan, an oral factor B inhibitor, demonstrated superior efficacy versus C5i in the phase 3 APPLY-PNH trial. This study evaluated the cost-utility of iptacopan versus C5i (eculizumab, ravulizumab, crovalimab) and pegcetacoplan (C3 inhibitor) in PNH patients previously treated with C5i.
METHODS: A semi-Markov model simulated the clinical course of PNH in health states for transfusion avoidance (with and without anemia), transfusion dependence, and death, over a lifetime horizon. Efficacy and safety inputs for iptacopan and C5 inhibitors (eculizumab and ravulizumab) were derived from APPLY-PNH. C5i parameters were used as a proxy for crovalimab, except for breakthrough hemolysis rates from COMMODORE-2. Efficacy and safety data for pegcetacoplan were based on PEGASUS. Health state utilities were derived from pivotal phase 3 trials of iptacopan. Direct medical costs, including drug list price, administration, prophylaxis, transfusion, and disease monitoring, were sourced from publicly available data and converted to USD. Model robustness was assessed using one-way sensitivity analysis and probabilistic sensitivity analysis.
RESULTS: Iptacopan dominated all comparators, achieving the highest total QALYs (12.028). Compared to eculizumab and ravulizumab, iptacopan provided gains of 2.030 QALYs and cost savings of USD 729,022 and USD 516,833, respectively. Against crovalimab and pegcetacoplan, iptacopan delivered QALY gains of 2.014 and 0.303 alongside savings of USD 150,516 and USD 69,761, respectively. Drug acquisition costs and utilities were key drivers of model outcomes.
CONCLUSIONS: In the Brazilian private healthcare system, iptacopan represented the most effective and cost-saving therapeutic option for PNH, reducing both its economic and logistical burden.
METHODS: A semi-Markov model simulated the clinical course of PNH in health states for transfusion avoidance (with and without anemia), transfusion dependence, and death, over a lifetime horizon. Efficacy and safety inputs for iptacopan and C5 inhibitors (eculizumab and ravulizumab) were derived from APPLY-PNH. C5i parameters were used as a proxy for crovalimab, except for breakthrough hemolysis rates from COMMODORE-2. Efficacy and safety data for pegcetacoplan were based on PEGASUS. Health state utilities were derived from pivotal phase 3 trials of iptacopan. Direct medical costs, including drug list price, administration, prophylaxis, transfusion, and disease monitoring, were sourced from publicly available data and converted to USD. Model robustness was assessed using one-way sensitivity analysis and probabilistic sensitivity analysis.
RESULTS: Iptacopan dominated all comparators, achieving the highest total QALYs (12.028). Compared to eculizumab and ravulizumab, iptacopan provided gains of 2.030 QALYs and cost savings of USD 729,022 and USD 516,833, respectively. Against crovalimab and pegcetacoplan, iptacopan delivered QALY gains of 2.014 and 0.303 alongside savings of USD 150,516 and USD 69,761, respectively. Drug acquisition costs and utilities were key drivers of model outcomes.
CONCLUSIONS: In the Brazilian private healthcare system, iptacopan represented the most effective and cost-saving therapeutic option for PNH, reducing both its economic and logistical burden.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE302
Topic
Economic Evaluation
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases