The Methodological Quality and Challenges in Conducting Economic Evaluations of Newborn Screening

The Methodological Quality and Challenges in Conducting Economic Evaluations of Newborn Screening: A Scoping Review

Cacciatore, P.
Visser, L.A.
Buyukkaramikli, N.
van der Ploeg, C.P.B.
van den Akker-van Marle, M.E.

2020

Introduction: Cost-effectiveness (CEA) and cost–utility analyses (CUA) have become popular types of economic evaluations (EE) used for evidence-based decision-making in healthcare resource allocation. Newborn screening programs (NBS) can have significant clinical benefits for society, and cost-effectiveness analysis may help to select the optimal strategy among different screening programs, including the no-screening option, on different conditions. These economic analyses of NBS, however, are hindered by several methodological challenges. This study explored the methodological quality in recent NBS economic evaluations and analyzed the main challenges and strategies adopted by researchers to deal with them. Methods: A scoping review was conducted according to PRISMA methodology to identify CEAs and CUAs of NBS. The methodological quality of the retrieved studies was assessed quantitatively using a specific guideline for the quality assessment of NBS economic evaluations, by calculating a general score for each EE. Challenges in the studies were then explored using thematic analysis as a qualitative synthesis approach. Results: Thirty-five studies met the inclusion criteria. The quantitative analysis showed that the methodological quality of NBS economic evaluations was heterogeneous. Lack of clear description of items related to results, discussion, and discounting were the most frequent flaws. Methodological challenges in performing EEs of neonatal screenings include the adoption of a long time horizon, the use of quality-adjusted life years as health outcome measure, and the assessment of costs beyond the screening interventions. Conclusions: The results of this review can support future economic evaluation research, aiding researchers to develop a methodological guidance to perform EEs aimed at producing solid results to inform decisions for resource allocation in neonatal screening. (This article belongs to the Special Issue Economic Evaluations of Newborn Screening: Methodological Considerations and Applications)

Economic evaluations
Newborn screening
Decision analysis
Methodology
Healthy for Life
Healthy Living
Life
CH - Child Health
ELSS - Earth, Life and Social Sciences

http://resolver.tudelft.nl/uuid:be0dbe9f-3fa9-449d-ac34-abbc56c32371

https://doi.org/10.3390/ijns6040094

884064

International Journal of Neonatal Screening, 6 (6), 94

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