Danicopan: A Dominant Option for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Residual Hemolytic Anemia in Spain
Author(s)
Miriam Castanyer, BPharm1, SANDRA MERINO, PhD2, Natalia Male, BSc3, Dario Rubio, Master in Health Economics4, MARIA CABALLERO HERRAIZ, BPharm5.
1alexion pharma spain, Barcelona, Spain, 2alexion pharma spain, BARCELONA, Spain, 3Alexion, Barcelona, Spain, 4Omakase Consulting, S.L, Madrid, Spain, 5Omakase Consulting, S.L, BARCELONA, Spain.
1alexion pharma spain, Barcelona, Spain, 2alexion pharma spain, BARCELONA, Spain, 3Alexion, Barcelona, Spain, 4Omakase Consulting, S.L, Madrid, Spain, 5Omakase Consulting, S.L, BARCELONA, Spain.
OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening blood disorder driven by complement-mediated intravascular hemolysis. Despite effective terminal complement inhibition, residual anemia often persists due to C3-mediated extravascular hemolysis. The study objective was to evaluate the cost-effectiveness (CE) of danicopan as an add-on therapy to the complement C5 inhibitors (C5i), ravulizumab and eculizumab, versus pegcetacoplan in patients with PNH with residual hemolytic anemia from a Spanish healthcare and societal perspective.
METHODS: A Markov state-transition model was developed with four mutually exclusive health states: Hb <9.5 g/dL (“Low Hb”), Hb ≥9.5 g/dL (“Medium Hb”), transfusion, and death. Within the “Low Hb”, “Medium Hb”, and “Transfusion states”, patients could experience a breakthrough hemolysis (BTH) event, after which a switch in treatment regimen was assumed. Health state transitions and model probabilities were based on patient-level data from the danicopan clinical trial. The model adopted a time horizon (up to 100 years of age; median 45.7 years), with annual discounting of 3.0% for costs and outcomes. Healthcare resource utilization and costs data were obtained from e-salud and BOTplus databases. Health state utilities were estimated using health-related quality of life (HRQoL) data from the ALPHA trial, and disutilities were applied for BTH, iron chelation therapy, and administration of pegcetacoplan and eculizumab. Price scenario analyses were conducted to assess the impact on CE outcomes.
RESULTS: Danicopan was dominant versus pegcetacoplan in the base case scenario with incremental QALY gains (0.352), from both, payer and societal perspectives. Applying a 30% discount to the list prices of pegcetacoplan and iC5, danicopan remained cost-effective (ICER: €7,564). When a 50% discount was applied to pegcetacoplan and iC5, danicopan maintained dominance when its list price was discounted by at least 29%.
CONCLUSIONS: Danicopan as add-on treatment to ravulizumab/ eculizumab is cost-effective in patients with PNH and residual hemolytic anemia across pricing scenarios
METHODS: A Markov state-transition model was developed with four mutually exclusive health states: Hb <9.5 g/dL (“Low Hb”), Hb ≥9.5 g/dL (“Medium Hb”), transfusion, and death. Within the “Low Hb”, “Medium Hb”, and “Transfusion states”, patients could experience a breakthrough hemolysis (BTH) event, after which a switch in treatment regimen was assumed. Health state transitions and model probabilities were based on patient-level data from the danicopan clinical trial. The model adopted a time horizon (up to 100 years of age; median 45.7 years), with annual discounting of 3.0% for costs and outcomes. Healthcare resource utilization and costs data were obtained from e-salud and BOTplus databases. Health state utilities were estimated using health-related quality of life (HRQoL) data from the ALPHA trial, and disutilities were applied for BTH, iron chelation therapy, and administration of pegcetacoplan and eculizumab. Price scenario analyses were conducted to assess the impact on CE outcomes.
RESULTS: Danicopan was dominant versus pegcetacoplan in the base case scenario with incremental QALY gains (0.352), from both, payer and societal perspectives. Applying a 30% discount to the list prices of pegcetacoplan and iC5, danicopan remained cost-effective (ICER: €7,564). When a 50% discount was applied to pegcetacoplan and iC5, danicopan maintained dominance when its list price was discounted by at least 29%.
CONCLUSIONS: Danicopan as add-on treatment to ravulizumab/ eculizumab is cost-effective in patients with PNH and residual hemolytic anemia across pricing scenarios
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE316
Topic
Economic Evaluation
Disease
Rare & Orphan Diseases