Journal Article

The cost-effectiveness analysis of newborn screening for inherited metabolic disorders in China using tandem mass spectrometry: a real-world evidence

Xiao, Dunming, Chen, Yingyao, Liu, Shimeng, Yuan, Jiaqi, Yang, Yi

2025

Overview

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. Key points • Text Text Text. •Research Significance: The study assesses the cost-effectiveness of MS/MS screening for IMDs in Chinese newborns, crucial for resource allocation and policy decisions. •Results Interpretation: MS/MS screening has higher costs but significantly better health outcomes, making it a cost-effective option below the GDP threshold. •Innovativeness: The study introduces a rigorous cost-effectiveness analysis to evaluate different screening strategies for IMDs. •Implications for Screening: The findings guide policymakers and healthcare providers towards a more effective and efficient newborn screening program for IMDs in China.

• Publisher: BioMed Central,BioMed Central Ltd,Springer Nature B.V,BMC
• Subject: Comparative analysis / Congenital diseases / Cost analysis / Cost benefit analysis / Cost-effectiveness analysis / Disease / Economic aspects / GDP / Gross Domestic Product / Health Administration / Health Economics / Health Services Research / Hospitalization / Hospitals / Infants (Newborn) / Inherited metabolic disorders / Mass spectrometry / Medical care / Medical records / Medical screening / Medicine / Medicine & Public Health / Metabolic disorders / Per capita / Pharmacoeconomics / Pharmacoeconomics and Health Outcomes / Phenylketonuria / Public Health / Quality management / Quality of Life Research / Scientific imaging / Screening / Tandem mass spectrometry / The Global South’s Long-Term Challenge of Sustainable Healthcare in the LMICs' Health Systems