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Cost-effectiveness of newborn screening for severe combined immunodeficiency: a systematic review
Rezwanul Rana1,2, Syed Afroz Keramat3, Moin Ahmed4
1Macquarie University Centre for the Health Economy, Macquarie University, Sydney, Australia.
Insights
Newborn bloodspot screening for severe combined immunodeficiency (SCID) is cost-effective. Early detection and treatment of SCID through universal screening improves infant health outcomes and life expectancy.
Area of Science:
- Immunology
- Genetics
- Public Health
Background:
- Severe combined immunodeficiency (SCID) is a rare genetic disorder causing severe infections and early childhood mortality.
- Newborn bloodspot screening (NBS) with the T-cell receptor excision circle assay offers early detection and treatment for SCID.
- Early intervention significantly improves quality of life and life expectancy for infants with SCID.
Purpose of the Study:
- To systematically review the cost-effectiveness of universal NBS for SCID.
- To synthesize evidence from economic evaluations of SCID screening programs.
Main Methods:
- Systematic literature search of multiple databases (MEDLINE, Embase, etc.) from January 2008 to March 2024.
- Inclusion of eight model-based economic evaluations (six cost-utility, two cost-effectiveness analyses).
- Data extraction on study characteristics, screening strategies, costs, health outcomes, and cost-effectiveness ratios.
Main Results:
- Universal NBS for SCID is highly likely to be cost-effective from both health system and societal perspectives.
- Incremental cost-effectiveness ratios ranged from $30,214 to $54,282 per quality-adjusted life-year gained (USD 2022).
- Evidence supports the value of population-based NBS for SCID.
Conclusions:
- Universal NBS for SCID represents good value for money, improving health outcomes.
- Early treatment of SCID is crucial and supported by screening.
- Further information on optimal treatment and associated costs is needed for informed policy decisions.
Abstract:
Severe combined immunodeficiency (SCID) is a rare genetic disorder that causes severe infections and death in early childhood. Newborn bloodspot screening (NBS) for SCID using the T-cell receptor excision circle assay can revolutionize the early detection and treatment of infants with SCID, leading to improved quality of life and life expectancy. This systematic review aimed to examine the cost-effectiveness of universal NBS for SCID. The MEDLINE, Embase, National Health Service (NHS) Economic Evaluation Database, Health Technology Assessment, Scopus, and EconLit databases were searched for studies of the NBS for SCID published between January 2008 and March 2024. A standardized data extraction form was used to gather pertinent data such as characteristics, design, perspective, screening strategies and costs, health outcomes, incremental cost-effectiveness ratios, and sources of uncertainty. Eight studies met our inclusion criteria: 6 cost-utility analyses and 2 cost-effectiveness analyses. All studies were model-based economic evaluations. These studies indicated that universal NBS for SCID is highly likely to demonstrate health system and societal cost-effectiveness. The incremental cost-effectiveness ratio per quality-adjusted life-year gained ranged from $30,214 to $54,282 (United States dollars 2022 value). Evidence suggests that early treatment of SCID is beneficial and that population-based NBS provides good value for the money. However, policymakers require better information about optimal treatment and treatment and screening costs to make informed decisions regarding competing healthcare priorities.
