Systematic review for economic evaluations on newborn screening for spinal muscular atrophy.

IF 3.2 4区 医学 Q2 CLINICAL NEUROLOGY
Alexander C Pace, Corrina Poon, Pranesh Chakraborty, Maryam Oskoui, Hugh McMillan, Alex Mackenzie, Jeff Round
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Abstract

ObjectiveEvaluate the quality and cost-effectiveness of economic evaluations of newborn screening (NBS) for Spinal Muscular Atrophy (SMA).MethodsA systematic review was conducted following Cochrane Handbook guidelines and PRISMA-S checklist. From 146 identified papers, 22 were screened for full-text, and 5 were included. Studies were evaluated for quality of reporting and transparency using the CHEERs and QHES checklists. Data was extracted to inform the review.ResultsFour economic evaluations on NBS for SMA with high reporting quality were identified. Each study employed a cost-utility analysis with similar model structures, using a decision tree for screening and a Markov model for treatment outcomes. They each compared NBS with treatment vs clinical diagnosis (no screening) with treatment. Although treatment protocols of each study varied due to differences in the strategies considered and availability of treatment. All studies included a societal perspective in their analysis and considered a lifetime horizon ranging from 30 months to 100 years. Early NBS with treatment was found to be more cost-effective than late treatment in all studies with ICER values ranging from £-117,541 to $714,000 per QALY. The wide range of ICER values are due to assumptions of long-term outcomes which are still largely unknown.ConclusionNBS with treatment was found to be cost-effective by all studies when compared to no NBS and late treatment. Although there is uncertainty around long term outcomes. Future research should focus on collecting long-term efficacy and safety data and evaluating the cost-effectiveness of pre-symptomatic treatment.

新生儿脊髓性肌萎缩症筛查经济评价的系统综述。
目的评价新生儿脊髓性肌萎缩症(SMA)筛查(NBS)经济评估的质量和成本-效果。方法按照Cochrane手册指南和PRISMA-S检查表进行系统评价。从146篇确定的论文中,22篇被筛选为全文,5篇被纳入。使用CHEERs和QHES清单评估研究报告的质量和透明度。数据被提取出来以供评价参考。结果鉴定出4个报告质量较高的国家统计局对SMA的经济评价。每项研究都采用了具有相似模型结构的成本效用分析,使用决策树进行筛选,使用马尔可夫模型进行治疗结果分析。他们分别比较了NBS治疗与临床诊断(无筛查)治疗。尽管每个研究的治疗方案因所考虑的策略和治疗的可用性的差异而有所不同。所有的研究都在分析中纳入了社会视角,并考虑了从30个月到100年不等的生命周期。在所有研究中,早期NBS治疗比晚期治疗更具成本效益,ICER值从每QALY -117,541英镑到714,000美元不等。ICER值的大范围是由于对长期结果的假设,而这些假设在很大程度上仍然未知。结论与不接受NBS治疗和晚期治疗相比,所有研究都发现NBS治疗具有成本效益。尽管长期结果存在不确定性。未来的研究应侧重于收集长期疗效和安全性数据,并评估症状前治疗的成本效益。
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来源期刊
Journal of neuromuscular diseases
Journal of neuromuscular diseases Medicine-Neurology (clinical)
CiteScore
5.10
自引率
6.10%
发文量
102
期刊介绍: The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic neuromuscular diseases (including muscular dystrophy, myasthenia gravis, spinal muscular atrophy, neuropathies, myopathies, myotonias and myositis). The journal publishes research reports, reviews, short communications, letters-to-the-editor, and will consider research that has negative findings. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.
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