Dunming Xiao, Jiaqi Yuan, Shimeng Liu, Yi Yang, Yingyao Chen
{"title":"The cost-effectiveness analysis of newborn screening for inherited metabolic disorders in China using tandem mass spectrometry: a real-world evidence.","authors":"Dunming Xiao, Jiaqi Yuan, Shimeng Liu, Yi Yang, Yingyao Chen","doi":"10.1186/s12962-025-00608-w","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Inherited metabolic disorders (IMDs) are a significant cause of morbidity and death among children. To determine the cost-effectiveness of newborn screening for IMDs using tandem mass spectrometry (MS/MS) compared to the non-screened group in China.</p><p><strong>Methods: </strong>We constructed a decision tree screening model based on the Chinese clinical path of tandem MS/MS screening for inherited metabolic disorders (IMDs) from the medical health system. This model simulated the mechanism of screening in the prevention and treatment of IMDs. The IMDs screening data was collected from Children's Hospital of Shanghai between 2010 and 2021. The Quality-adjusted life years (QALYs) and life expectancy were obtained from literature, while cost data was mainly sourced from hospital records and literature.</p><p><strong>Results: </strong>In the base-case analysis, the total lifetime cost per patient was higher for the MS/MS screened group at 1,000,452 Chinese Yuan (CNY) (USD 143,515), compared to 157,303 CNY (USD 22,565) for the non-screened group. The QALYs gained were 16.47 and 3.97 for the screened and non-screened groups, respectively. The incremental cost-effectiveness ratio (ICER) of the MS/MS screened group compared to the non-screened group was 67,417 CNY (USD 9,671) per QALY gained, which is under the threshold of 3 times per capita GDP of China in 2022 (242,928 CNY, USD 34,848). The benefit-cost ratio (BCR) was 4.23, which means that for every 1 CNY (USD 0.1434506) invested, a return of 4.23 CNY (USD 0.57) can be obtained. The probability of cost-effectiveness was 100% in the MS/MS screened group compared to the non-screened group, indicating that the results of the base case analysis were robust.</p><p><strong>Conclusion: </strong>Compared to the non-screened group, the MS/MS screened group incurs higher costs but also yields significantly greater QALY gains. Considering both the costs and benefits, the MS/MS screened group is an attractive cost-effective option at the current willingness-to-pay threshold for IMDs screening in China.</p>","PeriodicalId":47054,"journal":{"name":"Cost Effectiveness and Resource Allocation","volume":"23 1","pages":"3"},"PeriodicalIF":1.7000,"publicationDate":"2025-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cost Effectiveness and Resource Allocation","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1186/s12962-025-00608-w","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"HEALTH POLICY & SERVICES","Score":null,"Total":0}
引用次数: 0
Abstract
Background: Inherited metabolic disorders (IMDs) are a significant cause of morbidity and death among children. To determine the cost-effectiveness of newborn screening for IMDs using tandem mass spectrometry (MS/MS) compared to the non-screened group in China.
Methods: We constructed a decision tree screening model based on the Chinese clinical path of tandem MS/MS screening for inherited metabolic disorders (IMDs) from the medical health system. This model simulated the mechanism of screening in the prevention and treatment of IMDs. The IMDs screening data was collected from Children's Hospital of Shanghai between 2010 and 2021. The Quality-adjusted life years (QALYs) and life expectancy were obtained from literature, while cost data was mainly sourced from hospital records and literature.
Results: In the base-case analysis, the total lifetime cost per patient was higher for the MS/MS screened group at 1,000,452 Chinese Yuan (CNY) (USD 143,515), compared to 157,303 CNY (USD 22,565) for the non-screened group. The QALYs gained were 16.47 and 3.97 for the screened and non-screened groups, respectively. The incremental cost-effectiveness ratio (ICER) of the MS/MS screened group compared to the non-screened group was 67,417 CNY (USD 9,671) per QALY gained, which is under the threshold of 3 times per capita GDP of China in 2022 (242,928 CNY, USD 34,848). The benefit-cost ratio (BCR) was 4.23, which means that for every 1 CNY (USD 0.1434506) invested, a return of 4.23 CNY (USD 0.57) can be obtained. The probability of cost-effectiveness was 100% in the MS/MS screened group compared to the non-screened group, indicating that the results of the base case analysis were robust.
Conclusion: Compared to the non-screened group, the MS/MS screened group incurs higher costs but also yields significantly greater QALY gains. Considering both the costs and benefits, the MS/MS screened group is an attractive cost-effective option at the current willingness-to-pay threshold for IMDs screening in China.
期刊介绍：
Cost Effectiveness and Resource Allocation is an Open Access, peer-reviewed, online journal that considers manuscripts on all aspects of cost-effectiveness analysis, including conceptual or methodological work, economic evaluations, and policy analysis related to resource allocation at a national or international level. Cost Effectiveness and Resource Allocation is aimed at health economists, health services researchers, and policy-makers with an interest in enhancing the flow and transfer of knowledge relating to efficiency in the health sector. Manuscripts are encouraged from researchers based in low- and middle-income countries, with a view to increasing the international economic evidence base for health.
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