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首页> 外文期刊>BMC Pediatrics >Cost-effectiveness analysis of universal newborn screening for medium chain acyl-CoA dehydrogenase deficiency in France
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Cost-effectiveness analysis of universal newborn screening for medium chain acyl-CoA dehydrogenase deficiency in France

机译：法国普遍筛查中链酰基辅酶A脱氢酶缺乏症的成本效益分析

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Background Five diseases are currently screened on dried blood spots in France through the national newborn screening programme. Tandem mass spectrometry (MS/MS) is a technology that is increasingly used to screen newborns for an increasing number of hereditary metabolic diseases. Medium chain acyl-CoA dehydrogenase deficiency (MCADD) is among these diseases. We sought to evaluate the cost-effectiveness of introducing MCADD screening in France. Methods We developed a decision model to evaluate, from a societal perspective and a lifetime horizon, the cost-effectiveness of expanding the French newborn screening programme to include MCADD. Published and, where available, routine data sources were used. Both costs and health consequences were discounted at an annual rate of 4%. The model was applied to a French birth cohort. One-way sensitivity analyses and worst-case scenario simulation were performed. Results We estimate that MCADD newborn screening in France would prevent each year five deaths and the occurrence of neurological sequelae in two children under 5?years, resulting in a gain of 128 life years or 138 quality-adjusted life years (QALY). The incremental cost per year is estimated at €2.5 million, down to €1 million if this expansion is combined with a replacement of the technology currently used for phenylketonuria screening by MS/MS. The resulting incremental cost-effectiveness ratio (ICER) is estimated at €7 580/QALY. Sensitivity analyses indicate that while the results are robust to variations in the parameters, the model is most sensitive to the cost of neurological sequelae, MCADD prevalence, screening effectiveness and screening test cost. The worst-case scenario suggests an ICER of €72 000/QALY gained. Conclusions Although France has not defined any threshold for judging whether the implementation of a health intervention is an efficient allocation of public resources, we conclude that the expansion of the French newborn screening programme to MCADD would appear to be cost-effective. The results of this analysis have been used to produce recommendations for the introduction of universal newborn screening for MCADD in France.
机译：背景技术目前，法国通过国家新生儿筛查计划在干血斑上筛查了五种疾病。串联质谱（MS / MS）是一项越来越多的用于筛查新生儿遗传性代谢疾病的技术。这些疾病包括中链酰基辅酶A脱氢酶缺乏症（MCADD）。我们试图评估在法国引入MCADD筛查的成本效益。方法我们开发了一种决策模型，从社会角度和终身角度评估将法国新生儿筛查计划扩展到包括MCADD的成本效益。使用已发布的常规数据源（如果有的话）。成本和健康后果均以每年4％的比率折现。该模型已应用于法国出生队列。进行了单向敏感性分析和最坏情况模拟。结果我们估计，法国的MCADD新生儿筛查每年可预防2名5岁以下儿童中的5例死亡和神经系统后遗症的发生，从而增加128个生命年或138个质量调整生命年（QALY）。估计每年的增量成本为250万欧元，如果将此扩展与MS / MS替代当前用于苯丙酮尿症筛查的技术相结合，则可降低至100万欧元。由此产生的增量成本效益比（ICER）估计为7580欧元/ QALY。敏感性分析表明，尽管结果对参数变化具有鲁棒性，但该模型对神经系统后遗症的成本，MCADD患病率，筛查效果和筛查测试成本最为敏感。最坏的情况表明，ICER为每季度增加72000欧元。结论尽管法国尚未确定任何判断健康干预措施是否有效分配公共资源的门槛，但我们得出的结论是，法国新生儿筛查计划向MCADD的扩展似乎具有成本效益。该分析结果已被用于为在法国进行MCADD通用新生儿筛查提供建议。

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