Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency
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 p...
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| Format: | Article |
| Language: | English |
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MDPI AG
2022-07-01
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| Series: | International Journal of Neonatal Screening |
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| Online Access: | https://www.mdpi.com/2409-515X/8/3/44 |
| _version_ | 1797486948129964032 |
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| author | Sophy T. F. Shih Elena Keller Veronica Wiley Melanie Wong Michelle A. Farrar Georgina M. Chambers |
| author_facet | Sophy T. F. Shih Elena Keller Veronica Wiley Melanie Wong Michelle A. Farrar Georgina M. Chambers |
| author_sort | Sophy T. F. Shih |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-09T23:40:36Z |
| format | Article |
| id | doaj.art-2113b25f829940a7abd4d3c1d48b1545 |
| institution | Directory Open Access Journal |
| issn | 2409-515X |
| language | English |
| last_indexed | 2024-03-09T23:40:36Z |
| publishDate | 2022-07-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Neonatal Screening |
| spelling | doaj.art-2113b25f829940a7abd4d3c1d48b15452023-11-23T16:51:49ZengMDPI AGInternational Journal of Neonatal Screening2409-515X2022-07-01834410.3390/ijns8030044Economic Evaluation of Newborn Screening for Severe Combined ImmunodeficiencySophy T. F. Shih0Elena Keller1Veronica Wiley2Melanie Wong3Michelle A. Farrar4Georgina M. Chambers5Surveillance, Evaluation and Research Program, Kirby Institute, University of New South Wales, Sydney, NSW 2052, AustraliaNational Perinatal Epidemiology and Statistics Unit, Centre for Big Data Research in Health and School of Clinical Medicine, University of New South Wales, Sydney, NSW 2052, AustraliaNSW Newborn Screening Programme, Children’s Hospital Westmead, Westmead, NSW 2145, AustraliaDepartment of Allergy and Immunology, Children’s Hospital at Westmead, Westmead, NSW 2145, AustraliaDepartment of Neurology, Sydney Children’s Hospital, Randwick, Sydney, NSW 2031, AustraliaNational Perinatal Epidemiology and Statistics Unit, Centre for Big Data Research in Health and School of Clinical Medicine, University of New South Wales, Sydney, NSW 2052, AustraliaEvidence 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.https://www.mdpi.com/2409-515X/8/3/44SCIDnewborn screeningcost-effectivenessMarkov model |
| spellingShingle | Sophy T. F. Shih Elena Keller Veronica Wiley Melanie Wong Michelle A. Farrar Georgina M. Chambers Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency International Journal of Neonatal Screening SCID newborn screening cost-effectiveness Markov model |
| title | Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency |
| title_full | Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency |
| title_fullStr | Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency |
| title_full_unstemmed | Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency |
| title_short | Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency |
| title_sort | economic evaluation of newborn screening for severe combined immunodeficiency |
| topic | SCID newborn screening cost-effectiveness Markov model |
| url | https://www.mdpi.com/2409-515X/8/3/44 |
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