Cost-Utility Analysis of Pegunigalsidase Alfa Compared to Agalsidase Alfa and Agalsidase Beta for the Treatment of Adult Patients With Fabry Disease in Greece
Author(s)
Katerina Vellopoulou, MSc1, Katerina Lioliou, MSc1, Figaleia Karathanou, MSc1, Eirini Kiorpelidi, MSc2, Ilias Pyrnokokis, MSc2, Khashayar Azimpour, PhD3, Irene Koulinska, PhD4, Evangelia Dounousi, PhD5, Georgios Tsivgoulis, PhD6, Georgia Kourlaba, PhD7.
1ECONCARE LP, Athens, Greece, 2Chiesi Hellas SA, Alimos, Greece, 3Chiesi, Woodbridge, ON, Canada, 4Chiesi, Boston, MA, USA, 5Nephrology Department, University of Ioannina, Ioannina, Greece, 6Second Department of Neurology, School of Medicine, “Attikon” University Hospital, National and Kapodistrian University of Athens, Athens, Greece, 7Department of Nursing, School of Health Sciences, National and Kapodistrian University of Athens, Athens, Greece.
1ECONCARE LP, Athens, Greece, 2Chiesi Hellas SA, Alimos, Greece, 3Chiesi, Woodbridge, ON, Canada, 4Chiesi, Boston, MA, USA, 5Nephrology Department, University of Ioannina, Ioannina, Greece, 6Second Department of Neurology, School of Medicine, “Attikon” University Hospital, National and Kapodistrian University of Athens, Athens, Greece, 7Department of Nursing, School of Health Sciences, National and Kapodistrian University of Athens, Athens, Greece.
OBJECTIVES: Anderson-Fabry Disease (FD) is a rare disorder leading to significant morbidity. Enzyme replacement therapies (ERT) (agalsidase-alfa, agalsidase-beta, pegunigalsidase-alfa), along with migalastat have shown clinical benefits; however, their economic value remains a key consideration for payers. This research aims to assess the cost-utility of pegunigalsidase-alfa compared to agalsidase-alfa and agalsidase-beta for the treatment of adult patients with FD in Greece.
METHODS: A global cost-utility model was adapted to the Greek healthcare setting. To evaluate lifetime direct medical costs and health outcomes, expressed in quality-adjusted-life-years (QALYs), a Markov model, was employed for a hypothetical cohort of treatment-naïve and treatment-experienced adult patients with FD in Greece, from the perspective of National Organization for Health Services (EOPYY). Clinical inputs and utilities were sourced from pegunigalsidase-alfa completed clinical trials. Pegunigalsidase-alfa was compared to agalsidase-alfa and agalsidase-beta. Migalastat was excluded from this analysis since the comparable population is limited to patients with amenable mutations. Comparative clinical efficacy among ERTs was assessed using network meta-analysis and simulated treatment comparison, supporting the assumption of equivalent efficacy across ERTs. Healthcare resource use was informed by Greek clinical experts. Costs included drug acquisition, administration, health state management, and adverse events (AEs), valued using official sources and literature (euros,2024). Costs and QALYs were discounted at 3.5% annually. Incremental cost-effectiveness ratios were calculated. Deterministic and probabilistic sensitivity analyses (DSA, PSA) were performed to explore uncertainty.
RESULTS: Over lifetime, pegunigalsidase-alfa (€2,774,411) incurred €16,532 lower costs per patient compared to agalsidase-alfa (€2,790,943) and €386,467 lower costs compared to agalsidase-beta (€3,160,879). Pegunigalsidase-alfa yielded 0.090 more QALYs (11.278 vs. 11.187 for agalsidase-alfa/agalsidase-beta), driven by lower AEs disutilities in pegunigalsidase-alfa. DSA showed patient weight and administration costs as key drivers, while PSA confirmed results robustness.
CONCLUSIONS: Treatment with pegunigalsidase-alfa results in less costs and potentially improves health outcomes compared to currently used ERTs for adult patients with FD in Greece.
METHODS: A global cost-utility model was adapted to the Greek healthcare setting. To evaluate lifetime direct medical costs and health outcomes, expressed in quality-adjusted-life-years (QALYs), a Markov model, was employed for a hypothetical cohort of treatment-naïve and treatment-experienced adult patients with FD in Greece, from the perspective of National Organization for Health Services (EOPYY). Clinical inputs and utilities were sourced from pegunigalsidase-alfa completed clinical trials. Pegunigalsidase-alfa was compared to agalsidase-alfa and agalsidase-beta. Migalastat was excluded from this analysis since the comparable population is limited to patients with amenable mutations. Comparative clinical efficacy among ERTs was assessed using network meta-analysis and simulated treatment comparison, supporting the assumption of equivalent efficacy across ERTs. Healthcare resource use was informed by Greek clinical experts. Costs included drug acquisition, administration, health state management, and adverse events (AEs), valued using official sources and literature (euros,2024). Costs and QALYs were discounted at 3.5% annually. Incremental cost-effectiveness ratios were calculated. Deterministic and probabilistic sensitivity analyses (DSA, PSA) were performed to explore uncertainty.
RESULTS: Over lifetime, pegunigalsidase-alfa (€2,774,411) incurred €16,532 lower costs per patient compared to agalsidase-alfa (€2,790,943) and €386,467 lower costs compared to agalsidase-beta (€3,160,879). Pegunigalsidase-alfa yielded 0.090 more QALYs (11.278 vs. 11.187 for agalsidase-alfa/agalsidase-beta), driven by lower AEs disutilities in pegunigalsidase-alfa. DSA showed patient weight and administration costs as key drivers, while PSA confirmed results robustness.
CONCLUSIONS: Treatment with pegunigalsidase-alfa results in less costs and potentially improves health outcomes compared to currently used ERTs for adult patients with FD in Greece.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE307
Topic
Economic Evaluation, Health Technology Assessment
Topic Subcategory
Trial-Based Economic Evaluation
Disease
Rare & Orphan Diseases, Urinary/Kidney Disorders