COST-UTILITY ANALYSIS OF COMPLEMENT C5 INHIBITORS FOR PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) IN THAILAND - A PRELIMINARY ANALYSIS...
Author(s)
Pentai Taengutai, PharmD student, Sutinee Soopairin, PharmD, Parnnaphat Luksameesate, PhD, Suthira Taychakhoonavudh, BSc, MS, PhD, Chanthawat Patikorn, PharmD, PhD.
Faculty of Pharmaceutical Sciences Chulalongkorn University, Bangkok, Thailand.
Faculty of Pharmaceutical Sciences Chulalongkorn University, Bangkok, Thailand.
OBJECTIVES: This study aims to evaluate the cost-utility of complement C5 inhibitors for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) in Thailand.
METHODS: A six-state Markov model was developed to estimate the value for money of eculizumab or ravulizumab plus conventional therapy, compared to conventional therapy alone, in symptomatic PNH patients in Thailand. Lifetime costs and outcomes were estimated from a societal perspective and discounted at 3% annually. Model inputs, including transition probabilities, utilities, and costs, were obtained from targeted literature reviews, secondary data sources, and Thai-specific databases. Based on non-inferiority clinical trial evidence, treatment efficacy of eculizumab and ravulizumab were assumed to be equivalent. Outcomes included life-years, quality-adjusted life-years (QALYs), total costs, and incremental cost-effectiveness ratios (ICERs). Cost-effectiveness was then evaluated using Thai willingness-to-pay threshold of 160,000 THB/QALY.
RESULTS: Conventional therapy resulted in lifetime costs of 7.1 million THB for 23.31 life-years and 16.59 QALYs. Eculizumab and raizumab resulted in an incremental costs of 155.3 and 287.9 million THB, respectively. Both treatments yeildied 23.49 life-years and 19.28 QALYs. Compared with conventional therapy, adding eculizumab or ravulizumab yielded an additional of 2.69 QALYs and 0.12 life-years. With an ICER of 57.8 million THB/QALY for eculizumab and 107.1 million THB/QALY for ravulizumab. In addition, eculizumab and ravulizumab reduced lifetime model-predicted thromboembolic events from 4.39 to 0.35 events per patient, corresponding to 4.04 fewer events per patient (92.0% reduction).
CONCLUSIONS: Preliminary findings suggest that adding eculizumab or ravulizumab did not meet the Thai cost-effectiveness threshold. However, both treatments substantially reduced thromboembolic events compared with conventional therapy. Further evidence from budget impact and feasibility analyses are essential to inform reimbursement considerations and support decision-making regarding the potential inclusion of complement C5 inhibitors in a PNH benefit package in Thailand.
METHODS: A six-state Markov model was developed to estimate the value for money of eculizumab or ravulizumab plus conventional therapy, compared to conventional therapy alone, in symptomatic PNH patients in Thailand. Lifetime costs and outcomes were estimated from a societal perspective and discounted at 3% annually. Model inputs, including transition probabilities, utilities, and costs, were obtained from targeted literature reviews, secondary data sources, and Thai-specific databases. Based on non-inferiority clinical trial evidence, treatment efficacy of eculizumab and ravulizumab were assumed to be equivalent. Outcomes included life-years, quality-adjusted life-years (QALYs), total costs, and incremental cost-effectiveness ratios (ICERs). Cost-effectiveness was then evaluated using Thai willingness-to-pay threshold of 160,000 THB/QALY.
RESULTS: Conventional therapy resulted in lifetime costs of 7.1 million THB for 23.31 life-years and 16.59 QALYs. Eculizumab and raizumab resulted in an incremental costs of 155.3 and 287.9 million THB, respectively. Both treatments yeildied 23.49 life-years and 19.28 QALYs. Compared with conventional therapy, adding eculizumab or ravulizumab yielded an additional of 2.69 QALYs and 0.12 life-years. With an ICER of 57.8 million THB/QALY for eculizumab and 107.1 million THB/QALY for ravulizumab. In addition, eculizumab and ravulizumab reduced lifetime model-predicted thromboembolic events from 4.39 to 0.35 events per patient, corresponding to 4.04 fewer events per patient (92.0% reduction).
CONCLUSIONS: Preliminary findings suggest that adding eculizumab or ravulizumab did not meet the Thai cost-effectiveness threshold. However, both treatments substantially reduced thromboembolic events compared with conventional therapy. Further evidence from budget impact and feasibility analyses are essential to inform reimbursement considerations and support decision-making regarding the potential inclusion of complement C5 inhibitors in a PNH benefit package in Thailand.
Conference/Value in Health Info
2026-09, ISPOR Asia Pacific 2026, Bangkok, Thailand
Value in Health, Volume 55, Issue S1
Code
HTA23
Topic
Health Technology Assessment
Disease
SDC: Rare & Orphan Diseases