Exploring the Cost-Effectiveness of Newborn Screening for Metachromatic Leukodystrophy (MLD) in the UK.

IF 4 Q1 GENETICS & HEREDITY International Journal of Neonatal Screening Pub Date : 2024-06-26 DOI:10.3390/ijns10030045
Karen Bean, Simon A Jones, Anupam Chakrapani, Suresh Vijay, Teresa Wu, Heather Church, Charlotte Chanson, Andrew Olaye, Beckley Miller, Ivar Jensen, Francis Pang
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Abstract

Metachromatic leukodystrophy (MLD) is a fatal inherited lysosomal storage disease that can be detected through newborn bloodspot screening. The feasibility of the screening assay and the clinical rationale for screening for MLD have been previously demonstrated, so the aim of this study is to determine whether the addition of screening for MLD to the routine newborn screening program in the UK is a cost-effective use of National Health Service (NHS) resources. A health economic analysis from the perspective of the NHS and Personal Social Services was developed based on a decision-tree framework for each MLD subtype using long-term outcomes derived from a previously presented partitioned survival and Markov economic model. Modelling inputs for parameters related to epidemiology, test characteristics, screening and treatment costs were based on data from three major UK specialist MLD hospitals, structured expert opinion and published literature. Lifetime costs and quality-adjusted life years (QALYs) were discounted at 1.5% to account for time preference. Uncertainty associated with the parameter inputs was explored using sensitivity analyses. This health economic analysis demonstrates that newborn screening for MLD is a cost-effective use of NHS resources using a willingness-to-pay threshold appropriate to the severity of the disease; and supports the inclusion of MLD into the routine newborn screening programme in the UK.

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探索英国新生儿遗传性白营养不良症(MLD)筛查的成本效益。
变色性白质营养不良症(MLD)是一种致命的遗传性溶酶体储积症,可通过新生儿血斑筛查发现。筛查测定的可行性和筛查MLD的临床合理性此前已得到证实,因此本研究的目的是确定在英国常规新生儿筛查项目中增加MLD筛查是否是对国民健康服务(NHS)资源的一种具有成本效益的利用。从英国国家医疗服务体系(NHS)和个人社会服务机构的角度出发,利用以前提出的分区生存和马尔可夫经济模型得出的长期结果,在决策树框架的基础上对每种 MLD 亚型进行了健康经济分析。与流行病学、检测特征、筛查和治疗成本相关的参数的建模输入是基于英国三大 MLD 专科医院的数据、结构化的专家意见和已发表的文献。终生成本和质量调整生命年(QALYs)的贴现率为 1.5%,以考虑时间偏好。通过敏感性分析探讨了与参数输入相关的不确定性。这项卫生经济学分析表明,根据疾病的严重程度来确定支付意愿阈值,新生儿MLD筛查对NHS资源的使用具有成本效益;该分析还支持将MLD纳入英国的常规新生儿筛查计划。
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来源期刊
International Journal of Neonatal Screening
International Journal of Neonatal Screening Medicine-Pediatrics, Perinatology and Child Health
CiteScore
6.70
自引率
20.00%
发文量
56
审稿时长
11 weeks
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