Drug therapies for treatment of idiopathic pulmonary fibrosis: a systematic review, Bayesian network meta-analysis, and cost-effectiveness analysisResearch in context

Summary: Background: Idiopathic pulmonary fibrosis (IPF) is a progressive interstitial lung disease with poor prognosis and a high economic burden for individuals and healthcare resources. Studies of the costs associated with the efficiency of IPF medications are scarce. We aimed to conduct a netwo...

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Main Authors: Chunyang Zhao, Yan Yin, Chengrui Zhu, Min Zhu, Tianlong Ji, Zhonghao Li, Jiayi Cai
Format: Article
Language:English
Published: Elsevier 2023-07-01
Series:EClinicalMedicine
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2589537023002481
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author Chunyang Zhao
Yan Yin
Chengrui Zhu
Min Zhu
Tianlong Ji
Zhonghao Li
Jiayi Cai
author_facet Chunyang Zhao
Yan Yin
Chengrui Zhu
Min Zhu
Tianlong Ji
Zhonghao Li
Jiayi Cai
author_sort Chunyang Zhao
collection DOAJ
description Summary: Background: Idiopathic pulmonary fibrosis (IPF) is a progressive interstitial lung disease with poor prognosis and a high economic burden for individuals and healthcare resources. Studies of the costs associated with the efficiency of IPF medications are scarce. We aimed to conduct a network meta-analysis (NMA) and cost-effectiveness analysis to identify the optimum pharmacological strategy among all currently available IPF regimens. Methods: We first performed a systematic review and network meta-analysis. We searched eight databases for eligible randomised controlled trials (RCTs) published, in any language, between January 1, 1992 and July 31, 2022, that investigated the efficacy or tolerability (or both) of drug therapies for the treatment of IPF. The search was updated on February 1, 2023. Eligible RCTs were enrolled, with no restriction on dose, duration, or length of follow-up, if they included at least one of: all-cause mortality, acute exacerbation rate, disease progression rate, serious adverse events, and any adverse events under investigation. A subsequent Bayesian NMA within random-effects models was performed, followed by a cost-effectiveness analysis using the data obtained from our NMA, by developing a Markov model from the US payer's perspective. Assumptions were checked by deterministic and probabilistic sensitivity approaches to identify sensitive factors. We prospectively registered the protocol (CRD42022340590) in PROSPERO. Findings: 51 publications comprising 12,551 participants with IPF were analysed for the NMA, and the findings indicated that pirfenidone and N-acetylcysteine (NAC) + pirfenidone were the most efficacious and tolerable. The pharmacoeconomic analysis showed that NAC + pirfenidone was associated with the highest potentiality of being cost-effective at willingness-to-pay (WTP) thresholds of US$150,000 and $200,000, on the basis of quality-adjusted life years (QALYs), disability-adjusted life years (DALYs) and mortality, with the probability ranging from 53% to 92%. NAC was the minimum cost agent. Compared with placebo, NAC + pirfenidone improved effectiveness by increasing QALYs by 7.02, and reducing DALYs by 7.10 and deaths by 8.40, whilst raising overall costs by $516,894. Interpretation: This NMA and cost-effectiveness analysis suggests that NAC + pirfenidone is the most cost-effective option for treatment of IPF at WTP thresholds of $150,000 and $200,000. However, given that clinical practice guidelines have not addressed the application of this therapy, large well-designed and multicentre trials are warranted to provide a better picture of IPF management. Funding: None.
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spelling doaj.art-5b5f9c7f510940e1a40ecc0a459009232023-07-01T04:35:29ZengElsevierEClinicalMedicine2589-53702023-07-0161102071Drug therapies for treatment of idiopathic pulmonary fibrosis: a systematic review, Bayesian network meta-analysis, and cost-effectiveness analysisResearch in contextChunyang Zhao0Yan Yin1Chengrui Zhu2Min Zhu3Tianlong Ji4Zhonghao Li5Jiayi Cai6School of Pharmacy, China Medical University, Shenyang, China; Department of Pharmacy, The First Hospital of China Medical University, Shenyang, ChinaDepartment of Pulmonary and Critical Care Medicine, The First Hospital of China Medical University, Shenyang, ChinaDepartment of Critical Care Medicine, The First Hospital of China Medical University, Shenyang, ChinaSchool of Health Management, China Medical University, Shenyang, ChinaDepartment of Radiation Oncology, The First Hospital of China Medical University, Shenyang, ChinaChina Medical University-The Queen's University of Belfast Joint College, China Medical University, Shenyang, ChinaSchool of Pharmacy, China Medical University, Shenyang, China; Corresponding author. School of Pharmacy, China Medical University, No.77 Puhe Road, Shenyang, 110122, China.Summary: Background: Idiopathic pulmonary fibrosis (IPF) is a progressive interstitial lung disease with poor prognosis and a high economic burden for individuals and healthcare resources. Studies of the costs associated with the efficiency of IPF medications are scarce. We aimed to conduct a network meta-analysis (NMA) and cost-effectiveness analysis to identify the optimum pharmacological strategy among all currently available IPF regimens. Methods: We first performed a systematic review and network meta-analysis. We searched eight databases for eligible randomised controlled trials (RCTs) published, in any language, between January 1, 1992 and July 31, 2022, that investigated the efficacy or tolerability (or both) of drug therapies for the treatment of IPF. The search was updated on February 1, 2023. Eligible RCTs were enrolled, with no restriction on dose, duration, or length of follow-up, if they included at least one of: all-cause mortality, acute exacerbation rate, disease progression rate, serious adverse events, and any adverse events under investigation. A subsequent Bayesian NMA within random-effects models was performed, followed by a cost-effectiveness analysis using the data obtained from our NMA, by developing a Markov model from the US payer's perspective. Assumptions were checked by deterministic and probabilistic sensitivity approaches to identify sensitive factors. We prospectively registered the protocol (CRD42022340590) in PROSPERO. Findings: 51 publications comprising 12,551 participants with IPF were analysed for the NMA, and the findings indicated that pirfenidone and N-acetylcysteine (NAC) + pirfenidone were the most efficacious and tolerable. The pharmacoeconomic analysis showed that NAC + pirfenidone was associated with the highest potentiality of being cost-effective at willingness-to-pay (WTP) thresholds of US$150,000 and $200,000, on the basis of quality-adjusted life years (QALYs), disability-adjusted life years (DALYs) and mortality, with the probability ranging from 53% to 92%. NAC was the minimum cost agent. Compared with placebo, NAC + pirfenidone improved effectiveness by increasing QALYs by 7.02, and reducing DALYs by 7.10 and deaths by 8.40, whilst raising overall costs by $516,894. Interpretation: This NMA and cost-effectiveness analysis suggests that NAC + pirfenidone is the most cost-effective option for treatment of IPF at WTP thresholds of $150,000 and $200,000. However, given that clinical practice guidelines have not addressed the application of this therapy, large well-designed and multicentre trials are warranted to provide a better picture of IPF management. Funding: None.http://www.sciencedirect.com/science/article/pii/S2589537023002481Idiopathic pulmonary fibrosisDrug strategyBayesian network meta-analysisCost-effectiveness analysisAll-cause mortalityAcute exacerbation
spellingShingle Chunyang Zhao
Yan Yin
Chengrui Zhu
Min Zhu
Tianlong Ji
Zhonghao Li
Jiayi Cai
Drug therapies for treatment of idiopathic pulmonary fibrosis: a systematic review, Bayesian network meta-analysis, and cost-effectiveness analysisResearch in context
EClinicalMedicine
Idiopathic pulmonary fibrosis
Drug strategy
Bayesian network meta-analysis
Cost-effectiveness analysis
All-cause mortality
Acute exacerbation
title Drug therapies for treatment of idiopathic pulmonary fibrosis: a systematic review, Bayesian network meta-analysis, and cost-effectiveness analysisResearch in context
title_full Drug therapies for treatment of idiopathic pulmonary fibrosis: a systematic review, Bayesian network meta-analysis, and cost-effectiveness analysisResearch in context
title_fullStr Drug therapies for treatment of idiopathic pulmonary fibrosis: a systematic review, Bayesian network meta-analysis, and cost-effectiveness analysisResearch in context
title_full_unstemmed Drug therapies for treatment of idiopathic pulmonary fibrosis: a systematic review, Bayesian network meta-analysis, and cost-effectiveness analysisResearch in context
title_short Drug therapies for treatment of idiopathic pulmonary fibrosis: a systematic review, Bayesian network meta-analysis, and cost-effectiveness analysisResearch in context
title_sort drug therapies for treatment of idiopathic pulmonary fibrosis a systematic review bayesian network meta analysis and cost effectiveness analysisresearch in context
topic Idiopathic pulmonary fibrosis
Drug strategy
Bayesian network meta-analysis
Cost-effectiveness analysis
All-cause mortality
Acute exacerbation
url http://www.sciencedirect.com/science/article/pii/S2589537023002481
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