Cost–utility and budget impact analysis of tocilizumab for the treatment of refractory systemic juvenile idiopathic arthritis in Thailand

Objectives This study aimed to analyse the cost–utility and budget impact of adding tocilizumab to the standard treatment for patients with refractory systemic juvenile idiopathic arthritis (sJIA) in Thailand.Design Economic evaluation using a decision-analytical model.Setting Thailand.Participants...

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Main Authors: Nitichen Kittiratchakool, Disorn Kulpokin, Chonticha Chanjam, Soamarat Vilaiyuk, Sirirat Charuvanij, Gun Phongsamart, Parichat Khaosut, Manasita Tanya, Ratanavadee Nanagara, Sira Nantapaisarn, Pattara Leelahavarong
Format: Article
Language:English
Published: BMJ Publishing Group 2020-09-01
Series:BMJ Open
Online Access:https://bmjopen.bmj.com/content/10/9/e037588.full
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author Nitichen Kittiratchakool
Disorn Kulpokin
Chonticha Chanjam
Soamarat Vilaiyuk
Sirirat Charuvanij
Gun Phongsamart
Parichat Khaosut
Manasita Tanya
Ratanavadee Nanagara
Sira Nantapaisarn
Pattara Leelahavarong
author_facet Nitichen Kittiratchakool
Disorn Kulpokin
Chonticha Chanjam
Soamarat Vilaiyuk
Sirirat Charuvanij
Gun Phongsamart
Parichat Khaosut
Manasita Tanya
Ratanavadee Nanagara
Sira Nantapaisarn
Pattara Leelahavarong
author_sort Nitichen Kittiratchakool
collection DOAJ
description Objectives This study aimed to analyse the cost–utility and budget impact of adding tocilizumab to the standard treatment for patients with refractory systemic juvenile idiopathic arthritis (sJIA) in Thailand.Design Economic evaluation using a decision-analytical model.Setting Thailand.Participants Patients with refractory sJIA who were ≥2 years old.Methods The use of tocilizumab as an add-on therapy to standard treatment was compared with standard treatment alone. A simulated health state transition model was used to estimate the lifetime costs and health outcomes from a societal perspective. Direct medical costs were collected from tertiary hospital databases while direct non-medical costs were derived from interviews. Health-related quality of life (QoL) was measured using the proxy version of three-level EuroQol five-dimensional questionnaire (EQ-5D-3L). Future costs and outcomes were discounted at an annual rate of 3%. The base case population was patients aged 9.41 years old at refractory disease onset. The results were reported as incremental cost-effectiveness ratios (ICER) in US dollar (USD). One-way and probabilistic sensitivity analysis were conducted to investigate parameter uncertainty. The 5-year budget impact was estimated from a governmental perspective.Results The ICER of standard treatment plus tocilizumab was US$35 799 per quality-adjusted life-year (QALY) gained compared with standard treatment alone, which was not cost-effective at the threshold of US$5128 per QALY gained. The estimated 5 years budget impact was approximately US$4.8 million.Conclusions The use of standard treatment plus tocilizumab was not cost-effective in the Thai context, which has limited data. However, there is currently no second-line treatment for refractory sJIA in the Thai National List of Essential Medicines; thus, patients must receive higher doses of standard treatment which can cause many side effects. In contrast, tocilizumab showed obvious efficacy in clinical trials in improving treatment response and QoL. Therefore, the price of tocilizumab should be negotiated to reduce the financial impact on the healthcare system.
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issn 2044-6055
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spelling doaj-art-8ee604e121e544749921b3cf4375098a2025-01-06T14:35:11ZengBMJ Publishing GroupBMJ Open2044-60552020-09-0110910.1136/bmjopen-2020-037588Cost–utility and budget impact analysis of tocilizumab for the treatment of refractory systemic juvenile idiopathic arthritis in ThailandNitichen Kittiratchakool0Disorn Kulpokin1Chonticha Chanjam2Soamarat Vilaiyuk3Sirirat Charuvanij4Gun Phongsamart5Parichat Khaosut6Manasita Tanya7Ratanavadee Nanagara8Sira Nantapaisarn9Pattara Leelahavarong10Health Intervention and Technology Assessment Program, Nonthaburi, ThailandHealth Intervention and Technology Assessment Program, Nonthaburi, ThailandHealth Intervention and Technology Assessment Program, Nonthaburi, Thailand4Ped Rheum, Bangkok, ThailandDivision of Rheumatology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, ThailandDivision of Allergy, Immunology and Rheumatology, Department of Pediatrics, Queen Sirikit National Institute of Child Health, Bangkok, Thailand4King Chulalongkorn Memorial Hospital, Paediatric Rheumatology, Bangkok, United KingdomKK Women’s and Children’s Hospital, Rheumatology and Immunology, Singapore, SingaporeKhon Kaen University, Department of Medicine, Khon Kaen, ThailandDivision of Allergy, Immunology and Rheumatology, Department of Pediatrics, Faculty of Medicine, Thammasat University, Pathum Thani, ThailandHealth Intervention and Technology Assessment Program, Nonthaburi, ThailandObjectives This study aimed to analyse the cost–utility and budget impact of adding tocilizumab to the standard treatment for patients with refractory systemic juvenile idiopathic arthritis (sJIA) in Thailand.Design Economic evaluation using a decision-analytical model.Setting Thailand.Participants Patients with refractory sJIA who were ≥2 years old.Methods The use of tocilizumab as an add-on therapy to standard treatment was compared with standard treatment alone. A simulated health state transition model was used to estimate the lifetime costs and health outcomes from a societal perspective. Direct medical costs were collected from tertiary hospital databases while direct non-medical costs were derived from interviews. Health-related quality of life (QoL) was measured using the proxy version of three-level EuroQol five-dimensional questionnaire (EQ-5D-3L). Future costs and outcomes were discounted at an annual rate of 3%. The base case population was patients aged 9.41 years old at refractory disease onset. The results were reported as incremental cost-effectiveness ratios (ICER) in US dollar (USD). One-way and probabilistic sensitivity analysis were conducted to investigate parameter uncertainty. The 5-year budget impact was estimated from a governmental perspective.Results The ICER of standard treatment plus tocilizumab was US$35 799 per quality-adjusted life-year (QALY) gained compared with standard treatment alone, which was not cost-effective at the threshold of US$5128 per QALY gained. The estimated 5 years budget impact was approximately US$4.8 million.Conclusions The use of standard treatment plus tocilizumab was not cost-effective in the Thai context, which has limited data. However, there is currently no second-line treatment for refractory sJIA in the Thai National List of Essential Medicines; thus, patients must receive higher doses of standard treatment which can cause many side effects. In contrast, tocilizumab showed obvious efficacy in clinical trials in improving treatment response and QoL. Therefore, the price of tocilizumab should be negotiated to reduce the financial impact on the healthcare system.https://bmjopen.bmj.com/content/10/9/e037588.full
spellingShingle Nitichen Kittiratchakool
Disorn Kulpokin
Chonticha Chanjam
Soamarat Vilaiyuk
Sirirat Charuvanij
Gun Phongsamart
Parichat Khaosut
Manasita Tanya
Ratanavadee Nanagara
Sira Nantapaisarn
Pattara Leelahavarong
Cost–utility and budget impact analysis of tocilizumab for the treatment of refractory systemic juvenile idiopathic arthritis in Thailand
BMJ Open
title Cost–utility and budget impact analysis of tocilizumab for the treatment of refractory systemic juvenile idiopathic arthritis in Thailand
title_full Cost–utility and budget impact analysis of tocilizumab for the treatment of refractory systemic juvenile idiopathic arthritis in Thailand
title_fullStr Cost–utility and budget impact analysis of tocilizumab for the treatment of refractory systemic juvenile idiopathic arthritis in Thailand
title_full_unstemmed Cost–utility and budget impact analysis of tocilizumab for the treatment of refractory systemic juvenile idiopathic arthritis in Thailand
title_short Cost–utility and budget impact analysis of tocilizumab for the treatment of refractory systemic juvenile idiopathic arthritis in Thailand
title_sort cost utility and budget impact analysis of tocilizumab for the treatment of refractory systemic juvenile idiopathic arthritis in thailand
url https://bmjopen.bmj.com/content/10/9/e037588.full
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