Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency

Abstract

by Sophy T. F. Shih,Elena Keller,Veronica Wiley,Melanie Wong,Michelle A. Farrar andGeorgina M. Chambers
Int. J. Neonatal Screen. 2022, 8(3), 44; https://doi.org/10.3390/ijns8030044 - 20 Jul 2022
Cited by 8 | Viewed by 3548
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 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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13 pages, 250 KiB
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