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Challenges in Assessing the Cost-Effectiveness of Newborn Screening: The Example of Congenital Adrenal Hyperplasia

Scott D Grosse1, Guy Van Vliet2

  • 1National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA 30341, USA.

International Journal of Neonatal Screening
|November 26, 2020
PubMed

Insights

Evaluating the cost-effectiveness of newborn screening (NBS) is complex. Congenital adrenal hyperplasia (CAH) screening presents challenges due to varied methods and data, impacting policy decisions.

Area of Science:

  • Public Health
  • Health Economics
  • Genetics

Background:

  • Newborn screening (NBS) cost-effectiveness is challenging to generalize due to diverse included disorders and data limitations.
  • The influence of cost-effectiveness evidence on screening decisions for specific disorders remains unclear.
  • Congenital adrenal hyperplasia (CAH) screening serves as a pertinent case study due to the lack of global consensus on its inclusion in NBS panels.

Purpose of the Study:

  • To examine the methodological challenges in assessing the cost-effectiveness of newborn screening for congenital adrenal hyperplasia (CAH).
  • To provide insights for policymakers regarding the interpretation of economic evaluations for NBS programs.

Main Methods:

  • Review of published and unpublished cost-effectiveness analyses (CEAs) of CAH screening.
  • Analysis of variations in methods and data sources used in CEAs.
  • Examination of factors contributing to mixed findings in CAH screening CEAs, including health outcome estimation and cost differences between early and late diagnosis.

Main Results:

  • Cost-effectiveness analyses of CAH screening have produced inconsistent results.
  • Methodological differences, including data sources for health outcomes and costs, significantly impact CEA findings.
  • Between-country variations further complicate the generalization of CAH screening cost-effectiveness.

Conclusions:

  • Methodological heterogeneity and data limitations present significant challenges in determining the cost-effectiveness of NBS programs.
  • Understanding these challenges is crucial for conducting robust economic evaluations and informing policy decisions on NBS inclusion.
  • The case of CAH highlights the need for standardized methodologies and transparent reporting in NBS cost-effectiveness research.

Keywords:
congenital adrenal hyperplasiacost-effectivenesseconomic evaluationhealth policyneonatal screening

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