Challenges in Assessing the Cost-Effectiveness of Newborn Screening: The Example of Congenital Adrenal Hyperplasia
Open Access
- 25 October 2020
- journal article
- review article
- Published by MDPI AG in International Journal of Neonatal Screening
- Vol. 6 (4), 82
- https://doi.org/10.3390/ijns6040082
Abstract
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 Published and unpublished cost-effectiveness analyses of CAH screening have yielded mixed findings, largely due to differences in methods and data sources for estimating health outcomes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.Keywords
This publication has 100 references indexed in Scilit:
- Comparison of One-Tier and Two-Tier Newborn Screening Metrics for Congenital Adrenal HyperplasiaPEDIATRICS, 2012
- Cost-effectiveness analysis of universal newborn screening for medium chain acyl-CoA dehydrogenase deficiency in FranceBMC Pediatrics, 2012
- A Markov model to analyze cost-effectiveness of screening for severe combined immunodeficiency (SCID)Molecular Genetics and Metabolism, 2011
- Challenges in Health State Valuation in Paediatric Economic EvaluationPharmacoEconomics, 2011
- Health Status of Adults with Congenital Adrenal Hyperplasia: A Cohort Study of 203 PatientsJournal of Clinical Endocrinology & Metabolism, 2010
- Additional sensitivity data for newborn screening for congenital adrenal hyperplasia in WisconsinThe Journal of Pediatrics, 2006
- Mortality in patients with congenital adrenal hyperplasia: A cohort studyThe Journal of Pediatrics, 1998
- Newborn screening for congenital adrenal hyperplasia in New ZealandThe Journal of Pediatrics, 1995
- Cognitive and neuroradiological findings in congenital adrenal hyperplasiaPsychoneuroendocrinology, 1994
- Learning Disabilities in Children With Congenital Adrenal HyperplasiaJournal of Child Neurology, 1991