Danicopan in Addition to the Component 5 Inhibitor C5i Ravulizumab for the Treatment of Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Residual Hemolytic Anemia in Greece: A Cost Utility Analysis
Author(s)
Katerina Vellopoulou, MSc1, Stylianos Ravanidis, PhD1, Aggeliki Giannakopoulou, MSc2, Nikolas Kourkoulas, BSc, MD2, Alexandros Sfikas, PhD2, Nikolas Mathioudakis, PhD2, Georgia Kourlaba, PhD3.
1ECONCARE LP, Athens, Greece, 2ASTRAZENECA, Athens, Greece, 3Department of Nursing, School of Health Sciences, National & Kapodistrian University of Athens, Athens, Greece.
1ECONCARE LP, Athens, Greece, 2ASTRAZENECA, Athens, Greece, 3Department of Nursing, School of Health Sciences, National & Kapodistrian University of Athens, Athens, Greece.
OBJECTIVES: To assess the cost-utility of danicopan in addition to ravulizumab (danicopan/ravulizumab) compared with pegcetagoplan, for the treatment of adult patients with PNH with clinically significant extravascular hemolysis (i.e., residual hemolytic anemia) (PNH-EVH) in Greece.
METHODS: A cost-effectiveness Markov model (CEM) was adapted to estimate the costs and health outcomes of a hypothetical cohort of patients with PNH-EVH over a lifetime horizon, from the third-party payer perspective (EOPYY). It was adapted accordingly to depict the Greek clinical practice in PNH- EVH. Clinical data of danicopan/ravulizumab and pegcetagoplan in CEM were sourced from ALPHA and PEGASUS clinical trials, respectively. Healthcare resource use data were derived from literature and validated by Greek clinical experts. Drug costs, administration and management costs of breakthrough hemolysis (BTH), iron overload and adverse events were considered in the model. Unit costs were retrieved from official sources and literature (€,2024). Health outcomes were expressed in quality-adjusted life years (QALYs), as sourced from literature. All the future costs and outcomes are discounted at 3.5% per annum. Costs and effects of danicopan/ravulizumab vs pegcetagoplan were compared to assess the incremental cost-effectiveness ratio. A deterministic sensitivity analysis was undertaken to assess the parameters that mainly affect the analysis while a probabilistic sensitivity analysis (PSA) was applied to account for uncertainty in the model.
RESULTS: Treatment with danicopan/ravulizumab was associated with less costs (€6,202,168) compared with pegcetagoplan (€6,686,190), resulting in lower incremental costs (-€484,022) and in additional QALYs (0.476), indicating that it is a dominant treatment option. The proportion of BTH events for pegcetacoplan in the second treatment period and the cost of ravulizumab were the parameters that would mostly affect the results. PSA confirmed the robustness of the base case results.
CONCLUSIONS: Danicopan add-on treatment to ravulizumab is considered a dominant treatment option in adult patients with PNH-EVH in Greece compared with pegcetagoplan.
METHODS: A cost-effectiveness Markov model (CEM) was adapted to estimate the costs and health outcomes of a hypothetical cohort of patients with PNH-EVH over a lifetime horizon, from the third-party payer perspective (EOPYY). It was adapted accordingly to depict the Greek clinical practice in PNH- EVH. Clinical data of danicopan/ravulizumab and pegcetagoplan in CEM were sourced from ALPHA and PEGASUS clinical trials, respectively. Healthcare resource use data were derived from literature and validated by Greek clinical experts. Drug costs, administration and management costs of breakthrough hemolysis (BTH), iron overload and adverse events were considered in the model. Unit costs were retrieved from official sources and literature (€,2024). Health outcomes were expressed in quality-adjusted life years (QALYs), as sourced from literature. All the future costs and outcomes are discounted at 3.5% per annum. Costs and effects of danicopan/ravulizumab vs pegcetagoplan were compared to assess the incremental cost-effectiveness ratio. A deterministic sensitivity analysis was undertaken to assess the parameters that mainly affect the analysis while a probabilistic sensitivity analysis (PSA) was applied to account for uncertainty in the model.
RESULTS: Treatment with danicopan/ravulizumab was associated with less costs (€6,202,168) compared with pegcetagoplan (€6,686,190), resulting in lower incremental costs (-€484,022) and in additional QALYs (0.476), indicating that it is a dominant treatment option. The proportion of BTH events for pegcetacoplan in the second treatment period and the cost of ravulizumab were the parameters that would mostly affect the results. PSA confirmed the robustness of the base case results.
CONCLUSIONS: Danicopan add-on treatment to ravulizumab is considered a dominant treatment option in adult patients with PNH-EVH in Greece compared with pegcetagoplan.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE317
Topic
Economic Evaluation, Health Technology Assessment
Topic Subcategory
Trial-Based Economic Evaluation
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)