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01-05-2019 | Immunodeficiency | Original Article

Cost-effectiveness of newborn screening for severe combined immunodeficiency

Authors: Catharina P. B. Van der Ploeg, Maartje Blom, Robbert G. M. Bredius, Mirjam van der Burg, Peter C. J. I. Schielen, Paul H. Verkerk, M. Elske Van den Akker-van Marle

Published in: European Journal of Pediatrics | Issue 5/2019

Abstract

Severe combined immunodeficiency (SCID) is a condition that often results in severe infections and death at young age. Early detection shortly after birth, followed by treatment before infections occur, largely increases the chances of survival. As the incidence of SCID is low, assessing cost-effectiveness of adding screening for SCID to the newborn screening program is relevant for decision making. Lifetime costs and effects of newborn screening for SCID were compared to a situation without screening in the Netherlands in a decision analysis model. Model parameters were based on literature and expert opinions. Sensitivity analyses were performed. Due to earlier detection, the number of deaths due to SCID per 100,000 children was assessed to decrease from 0.57 to 0.23 and a number of 11.7 quality adjusted life-years (QALYs) gained was expected. Total yearly healthcare costs, including costs of screening, diagnostics, and treatment, were €390,800 higher in a situation with screening compared to a situation without screening, resulting in a cost-utility ratio of €33,400 per QALY gained.

Conclusion: Newborn screening for SCID might be cost-effective. However, there is still a lot of uncertainty around the cost-effectiveness estimate. Pilot screening projects are warranted to obtain more accurate estimates for the European situation.

What is Known:
• Severe combined immunodeficiency (SCID) is a condition that often results in severe infections and death at a young age. • As the incidence of SCID is low, assessing cost-effectiveness of adding screening for SCID to the newborn screening program is needed.
What is New: • Newborn screening for SCID is expected to reduce mortality from 0.57 to 0.23 per 100,000 children at additional healthcare costs of €390,800. The cost-utility ratio is €33,400 per QALY gained. • Due to large uncertainty around cost-effectiveness estimates, pilot screening projects are warranted for Europe.

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Title: Cost-effectiveness of newborn screening for severe combined immunodeficiency
Authors: Catharina P. B. Van der Ploeg
Maartje Blom
Robbert G. M. Bredius
Mirjam van der Burg
Peter C. J. I. Schielen
Paul H. Verkerk
M. Elske Van den Akker-van Marle
Publication date: 01-05-2019
Publisher: Springer Berlin Heidelberg
Keywords: Immunodeficiency
Expert Opinion
Neonatal Screening
Expert Opinion
Published in: European Journal of Pediatrics / Issue 5/2019
Print ISSN: 0340-6199
Electronic ISSN: 1432-1076
DOI: https://doi.org/10.1007/s00431-019-03346-3

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