Special Issue "Economic Evaluations of Newborn Screening: Methodological Considerations and Applications"

A special issue of International Journal of Neonatal Screening (ISSN 2409-515X).

Deadline for manuscript submissions: closed (30 September 2020).

Special Issue Editors

Dr. M. Elske Van den Akker-van Marle
E-Mail Website
Guest Editor
Department of Biomedical Data Sciences, section Medical Decision Making, Leiden University Medical Center (LUMC), P.O. Box 9600, 2300 RC Leiden, The Netherlands
Interests: newborn screening program; economic evaluations; cost-effectiveness/cost-utility; quality of life
Dr. Catharina P.B. Van der Ploeg
E-Mail Website
Guest Editor
Department of Child Health, TNO, P.O. Box 3005, 2301 DA Leiden, the Netherlands
Interests: screening programs; newborn screening; monitoring and evaluation; economic evaluation; cost-effectiveness/cost-utility

Special Issue Information

Dear Colleagues,

Economic evaluations of healthcare interventions inform incremental costs and effects of interventions compared to a relevant comparator, such as standard care. This information can be used by policymakers to decide which interventions to fund or implement in practice. Especially in newborn screening (NBS), frequently with respect to rare diseases, assessing the cost-effectiveness of adding a screening to the NBS program is relevant for decision making. However, publications on the cost-effectiveness of NBS are relatively scarce. This may be due to methodological difficulties that limit the applicability of standard economic evaluation methods. These include the absence of randomized controlled trials to estimate the effectiveness of NBS, limited data due to rareness of diseases, measuring QALYs in a pediatric population, assessing long-term (lifelong) outcomes and costs, and quantifying impact on reproductive decisions.

For this Special Issue on “Economic Evaluations of Newborn Screening: Methodological Considerations and Applications”, we welcome papers on methodological issues around economic evaluations of NBS and papers reporting an economic evaluation of NBS.

Dr. M. Elske Van den Akker-van Marle
Dr C.P.B. (Kitty) van der Ploeg
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All papers will be peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. International Journal of Neonatal Screening is an international peer-reviewed open access quarterly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 800 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • newborn screening
  • economic evaluation
  • cost-effectiveness/cost-utility
  • quality of life

Published Papers (3 papers)

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Research

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Open AccessArticle
The Methodological Quality and Challenges in Conducting Economic Evaluations of Newborn Screening: A Scoping Review
Int. J. Neonatal Screen. 2020, 6(4), 94; https://doi.org/10.3390/ijns6040094 - 23 Nov 2020
Viewed by 767
Abstract
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 [...] Read more.
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. 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. 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. 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. Full article
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Open AccessArticle
The Cost-Effectiveness of Expanding the UK Newborn Bloodspot Screening Programme to Include Five Additional Inborn Errors of Metabolism
Int. J. Neonatal Screen. 2020, 6(4), 93; https://doi.org/10.3390/ijns6040093 - 20 Nov 2020
Viewed by 807
Abstract
Glutaric aciduria type 1, homocystinuria, isovaleric acidaemia, long-chain hydroxyacyl CoA dehydrogenase deficiency and maple syrup urine disease are all inborn errors of metabolism that can be detected through newborn bloodspot screening. This evaluation was undertaken in 2013 to provide evidence to the UK [...] Read more.
Glutaric aciduria type 1, homocystinuria, isovaleric acidaemia, long-chain hydroxyacyl CoA dehydrogenase deficiency and maple syrup urine disease are all inborn errors of metabolism that can be detected through newborn bloodspot screening. This evaluation was undertaken in 2013 to provide evidence to the UK National Screening Committee for the cost-effectiveness of including these five conditions in the UK Newborn Bloodspot Screening Programme. A decision-tree model with lifetable estimates of outcomes was built with the model structure and parameterisation informed by a systematic review and expert clinical judgment. A National Health Service/Personal Social Services perspective was used, and lifetime costs and quality-adjusted life years (QALYs) were discounted at 1.5%. Uncertainty in the results was explored using expected value of perfect information analysis methods together with a sensitivity analysis using the screened incidence rate in the UK from 2014 to 2018. The model estimates that screening for all the conditions is more effective and cost saving when compared to not screening for each of the conditions, and the results were robust to the updated incidence rates. The key uncertainties included the sensitivity and specificity of the screening test and the estimated costs and QALYs. Full article
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Review

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Open AccessReview
Challenges in Assessing the Cost-Effectiveness of Newborn Screening: The Example of Congenital Adrenal Hyperplasia
Int. J. Neonatal Screen. 2020, 6(4), 82; https://doi.org/10.3390/ijns6040082 - 25 Oct 2020
Cited by 1 | Viewed by 887
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 [...] Read more.
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. Full article
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