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Economic Evaluations of Newborn Screening: Methodological Considerations and Applications
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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 (31 December 2021) | Viewed by 18213

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 submissions that pass pre-check are 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 1600 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 (6 papers)

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Research

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15 pages, 2556 KiB  
Article
Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency
by Sophy T. F. Shih, Elena Keller, Veronica Wiley, Melanie Wong, Michelle A. Farrar and Georgina M. Chambers
Int. J. Neonatal Screen. 2022, 8(3), 44; https://doi.org/10.3390/ijns8030044 - 20 Jul 2022
Cited by 5 | Viewed by 3037
Abstract
Evidence on the cost-effectiveness of newborn screening (NBS) for severe combined immunodeficiency (SCID) in the Australian policy context is lacking. In this study, a pilot population-based screening program in Australia was used to model the cost-effectiveness of NBS for SCID from the government [...] Read more.
Evidence on the cost-effectiveness of newborn screening (NBS) for severe combined immunodeficiency (SCID) in the Australian policy context is lacking. In this study, a pilot population-based screening program in Australia was used to model the cost-effectiveness of NBS for SCID from the government perspective. Markov cohort simulations were nested within a decision analytic model to compare the costs and quality-adjusted life-years (QALYs) over a time horizon of 5 and 60 years for two strategies: (1) NBS for SCID and treat with early hematopoietic stem cell transplantation (HSCT); (2) no NBS for SCID and treat with late HSCT. Incremental costs were compared to incremental QALYs to calculate the incremental cost-effectiveness ratios (ICER). Sensitivity analyses were performed to assess the model uncertainty and identify key parameters impacting on the ICER. In the long-term over 60 years, universal NBS for SCID would gain 10 QALYs at a cost of US $0.3 million, resulting in an ICER of US$33,600/QALY. Probabilistic sensitivity analysis showed that more than half of the simulated ICERs were considered cost-effective against the common willingness-to-pay threshold of A$50,000/QALY (US$35,000/QALY). In the Australian context, screening for SCID should be introduced into the current NBS program from both clinical and economic perspectives. Full article
(This article belongs to the Special Issue Economic Evaluations of Newborn Screening: Methodological Considerations and Applications)
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9 pages, 965 KiB  
Article
Delivering Positive Newborn Screening Results: Cost Analysis of Existing Practice versus Innovative, Co-Designed Strategies from the ReSPoND Study
by Francesco Fusco, Jane Chudleigh, Pru Holder, James R. Bonham, Kevin W. Southern, Alan Simpson, Louise Moody, Ellinor K. Olander, Holly Chinnery and Stephen Morris
Int. J. Neonatal Screen. 2022, 8(1), 19; https://doi.org/10.3390/ijns8010019 - 14 Mar 2022
Cited by 1 | Viewed by 2301
Abstract
Although the communication pathways of Newborn Bloodspot Screening (NBS) are a delicate task, these pathways vary across different conditions and are often not evidence-based. The ReSPoND interventions were co-designed by healthcare professionals alongside parents who had received a positive NBS result for their [...] Read more.
Although the communication pathways of Newborn Bloodspot Screening (NBS) are a delicate task, these pathways vary across different conditions and are often not evidence-based. The ReSPoND interventions were co-designed by healthcare professionals alongside parents who had received a positive NBS result for their child. To calculate the cost of these co-designed strategies and the existing communication pathways, we interviewed 71 members of the clinical and laboratory staff of the 13 English NBS laboratories in the English National Health Service. Therefore, a scenario analysis was used to compare the cost of the existing communication pathways to the co-designed strategies delivered by (i) home-visits and (ii) telecommunications. On average, the existing communication pathway cost £447.08 per infant (range: £237.12 to £628.51) or £234,872.75 (£3635.99 to £1,932,986.23) nationally. Implementing the new interventions relying on home-visits exclusively would cost on average £521.62 (£312.84 to £646.39) per infant and £297,816.03 (£4506.37 to £2,550,284.64) nationally, or £447.19 (£235.79 to £552.03) and £231,342.40 (£3923.7 to £1,922,192.22) if implemented via teleconsultations, respectively. The new strategies delivered are not likely to require additional resources compared with current practice. Further research is needed to investigate whether this investment represents good value for money for the NHS budget. Full article
(This article belongs to the Special Issue Economic Evaluations of Newborn Screening: Methodological Considerations and Applications)
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13 pages, 263 KiB  
Article
Economic Evaluation of Different Screening Strategies for Severe Combined Immunodeficiency Based on Real-Life Data
by M. Elske van den Akker-van Marle, Maartje Blom, Mirjam van der Burg, Robbert G. M. Bredius and Catharina P. B. Van der Ploeg
Int. J. Neonatal Screen. 2021, 7(3), 60; https://doi.org/10.3390/ijns7030060 - 15 Sep 2021
Cited by 7 | Viewed by 2213
Abstract
Although several countries have adopted severe combined immunodeficiency (SCID) into their newborn screening (NBS) program, other countries are still in the decision process of adding this disorder in their program and finding the appropriate screening strategy. This decision may be influenced by the [...] Read more.
Although several countries have adopted severe combined immunodeficiency (SCID) into their newborn screening (NBS) program, other countries are still in the decision process of adding this disorder in their program and finding the appropriate screening strategy. This decision may be influenced by the cost(-effectiveness) of these screening strategies. In this study, the cost(-effectiveness) of different NBS strategies for SCID was estimated based on real-life data from a prospective implementation study in the Netherlands. The cost of testing per child for SCID was estimated at EUR 6.36. The cost of diagnostics after screen-positive results was assessed to vary between EUR 985 and 8561 per child dependent on final diagnosis. Cost-effectiveness ratios varied from EUR 41,300 per QALY for the screening strategy with T-cell receptor excision circle (TREC) ≤ 6 copies/punch to EUR 44,100 for the screening strategy with a cut-off value of TREC ≤ 10 copies/punch. The analysis based on real-life data resulted in higher costs, and consequently in less favorable cost-effectiveness estimates than analyses based on hypothetical data, indicating the need for verifying model assumptions with real-life data. The comparison of different screening strategies suggest that strategies with a lower number of referrals, e.g., by distinguishing between urgent and less urgent referrals, are favorable from an economic perspective. Full article
(This article belongs to the Special Issue Economic Evaluations of Newborn Screening: Methodological Considerations and Applications)
20 pages, 679 KiB  
Article
The Methodological Quality and Challenges in Conducting Economic Evaluations of Newborn Screening: A Scoping Review
by Pasquale Cacciatore, Laurenske A. Visser, Nasuh Buyukkaramikli, Catharina P. B. van der Ploeg and M. Elske van den Akker-van Marle
Int. J. Neonatal Screen. 2020, 6(4), 94; https://doi.org/10.3390/ijns6040094 - 23 Nov 2020
Cited by 6 | Viewed by 2901
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
(This article belongs to the Special Issue Economic Evaluations of Newborn Screening: Methodological Considerations and Applications)
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16 pages, 1316 KiB  
Article
The Cost-Effectiveness of Expanding the UK Newborn Bloodspot Screening Programme to Include Five Additional Inborn Errors of Metabolism
by Alice Bessey, James Chilcott, Abdullah Pandor and Suzy Paisley
Int. J. Neonatal Screen. 2020, 6(4), 93; https://doi.org/10.3390/ijns6040093 - 20 Nov 2020
Cited by 12 | Viewed by 3428
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
(This article belongs to the Special Issue Economic Evaluations of Newborn Screening: Methodological Considerations and Applications)
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Review

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19 pages, 330 KiB  
Review
Challenges in Assessing the Cost-Effectiveness of Newborn Screening: The Example of Congenital Adrenal Hyperplasia
by Scott D. Grosse and Guy Van Vliet
Int. J. Neonatal Screen. 2020, 6(4), 82; https://doi.org/10.3390/ijns6040082 - 25 Oct 2020
Cited by 11 | Viewed by 3529
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
(This article belongs to the Special Issue Economic Evaluations of Newborn Screening: Methodological Considerations and Applications)
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