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Economic Evaluation of Different Screening Strategies for Severe Combined Immunodeficiency Based on Real-Life Data

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 th...

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Autores principales: van den Akker-van Marle, M. Elske, Blom, Maartje, van der Burg, Mirjam, Bredius, Robbert G. M., Van der Ploeg, Catharina P. B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8482221/
https://www.ncbi.nlm.nih.gov/pubmed/34564080
http://dx.doi.org/10.3390/ijns7030060
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author van den Akker-van Marle, M. Elske
Blom, Maartje
van der Burg, Mirjam
Bredius, Robbert G. M.
Van der Ploeg, Catharina P. B.
author_facet van den Akker-van Marle, M. Elske
Blom, Maartje
van der Burg, Mirjam
Bredius, Robbert G. M.
Van der Ploeg, Catharina P. B.
author_sort van den Akker-van Marle, M. Elske
collection PubMed
description 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.
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spelling pubmed-84822212021-10-01 Economic Evaluation of Different Screening Strategies for Severe Combined Immunodeficiency Based on Real-Life Data van den Akker-van Marle, M. Elske Blom, Maartje van der Burg, Mirjam Bredius, Robbert G. M. Van der Ploeg, Catharina P. B. Int J Neonatal Screen Article 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. MDPI 2021-09-15 /pmc/articles/PMC8482221/ /pubmed/34564080 http://dx.doi.org/10.3390/ijns7030060 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
van den Akker-van Marle, M. Elske
Blom, Maartje
van der Burg, Mirjam
Bredius, Robbert G. M.
Van der Ploeg, Catharina P. B.
Economic Evaluation of Different Screening Strategies for Severe Combined Immunodeficiency Based on Real-Life Data
title Economic Evaluation of Different Screening Strategies for Severe Combined Immunodeficiency Based on Real-Life Data
title_full Economic Evaluation of Different Screening Strategies for Severe Combined Immunodeficiency Based on Real-Life Data
title_fullStr Economic Evaluation of Different Screening Strategies for Severe Combined Immunodeficiency Based on Real-Life Data
title_full_unstemmed Economic Evaluation of Different Screening Strategies for Severe Combined Immunodeficiency Based on Real-Life Data
title_short Economic Evaluation of Different Screening Strategies for Severe Combined Immunodeficiency Based on Real-Life Data
title_sort economic evaluation of different screening strategies for severe combined immunodeficiency based on real-life data
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8482221/
https://www.ncbi.nlm.nih.gov/pubmed/34564080
http://dx.doi.org/10.3390/ijns7030060
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