评估新生儿筛查成本效益的挑战：以先天性肾上腺皮质增生症为例。
Challenges in Assessing the Cost-Effectiveness of Newborn Screening: The Example of Congenital Adrenal Hyperplasia.
作者信息
Grosse Scott D, Van Vliet Guy
机构信息
National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA 30341, USA.
Endocrinology Service and Research Center of the Sainte-Justine Hospital and Department of Pediatrics, University of Montreal, Montreal, QC H3T 1C5, Canada.
出版信息
Int J Neonatal Screen. 2020 Oct 25;6(4):82. doi: 10.3390/ijns6040082.
Generalizing about the cost-effectiveness of newborn screening (NBS) is difficult due to the heterogeneity of disorders included in NBS panels, along with data limitations. Furthermore, it is unclear to what extent evidence about cost-effectiveness should influence decisions to screen for specific disorders. Screening newborns for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency can serve as a useful test case, since there is no global consensus on whether CAH should be part of NBS panels. Published and unpublished cost-effectiveness analyses of CAH screening have yielded mixed findings, largely due to differences in methods and data sources for estimating health outcomes and associated costs of early versus late diagnosis as well as between-country differences. Understanding these methodological challenges can help inform future analyses and could also help interested policymakers interpret the results of economic evaluations.
由于新生儿筛查（NBS）项目所包含疾病的异质性以及数据限制，很难对新生儿筛查的成本效益进行概括。此外，关于成本效益的证据应在多大程度上影响针对特定疾病进行筛查的决策尚不清楚。对因21-羟化酶缺乏导致的先天性肾上腺皮质增生症（CAH）进行新生儿筛查可以作为一个有用的测试案例，因为对于CAH是否应纳入NBS项目，全球尚未达成共识。已发表和未发表的CAH筛查成本效益分析结果不一，这主要是由于在估计健康结果以及早期与晚期诊断的相关成本方面，方法和数据来源存在差异，以及国家之间的差异。了解这些方法学挑战有助于为未来的分析提供信息，也有助于感兴趣的政策制定者解读经济评估的结果。
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