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Published on: December 6, 2014
Fiscal implications of newborn screening in the diagnosis of severe combined immunodeficiency
Catherine Kubiak1, Soma Jyonouchi2, Caroline Kuo3
1Department of Pediatrics, Division of Allergy and Immunology, Morsani College of Medicine, University of South Florida, Tampa, Fla.
Insights
Early screening for severe combined immunodeficiency (SCID) through newborn screening (NBS) significantly reduces treatment costs. This cost-effectiveness supports expanding SCID screening nationwide.
Area of Science:
- Medical Genetics
- Pediatric Immunology
- Health Economics
Background:
- Newborn screening (NBS) identifies 31 conditions, but state-specific implementation varies.
- Severe combined immunodeficiency (SCID) is fatal by age 2 if untreated; hematopoietic stem cell transplantation is curative if performed early.
- Implementation costs can be a barrier to adding SCID screening to NBS panels.
Purpose of the Study:
- To conduct a retrospective economic analysis comparing the cost-effectiveness of early versus late treatment for SCID.
- To evaluate the financial impact of SCID screening on newborn healthcare systems.
Main Methods:
- Retrospective economic analysis over 5 years at 3 centers.
- Comparison of total charges for early (<3.5 months) versus late (≥3.5 months) SCID treatment.
Main Results:
- Late SCID treatment costs were 4 times higher than early treatment ($1.43M vs $365,785).
- Intensive care costs were over 5 times higher for late treatment ($350,252 vs $66,379).
- Operating room and anesthesia charges were approximately 4 times higher for late treatment ($57,105 vs $15,885).
Conclusions:
- Early SCID treatment is significantly more cost-effective than late treatment.
- The economic benefits provide a strong rationale for expanding SCID screening in NBS programs.
- Implementing SCID screening can lead to substantial healthcare cost savings.
Abstract:
In the United States, newborn screening (NBS) is currently recommended for identification of 31 debilitating and potentially fatal conditions. However, individual states determine which of the recommended conditions are screened. The addition of severe combined immunodeficiency (SCID) screening to the recommended NBS panel has been fully instituted by 18 states, with another 11 states piloting programs or planning to begin screening in 2014. Untreated, SCID is uniformly fatal by 2 years of age. Hematopoietic stem cell transplantation usually is curative, but the success rate depends on the age at which the procedure is performed. Short-term implementation costs may be a barrier to adding SCID to states' NBS panels. A retrospective economic analysis was performed to determine the cost-effectiveness of NBS for early (<3.5 months) versus late (≥3.5 months) treatment of children with SCID at 3 centers over 5 years. The mean total charges at these centers for late treatment were 4 times greater than early treatment ($1.43 million vs $365,785, respectively). Mean charges for intensive care treatments were >5 times higher ($350,252 vs $66,379), and operating room-anesthesia charges were approximately 4 times higher ($57,105 vs $15,885). The cost-effectiveness of early treatment for SCID provides a strong economic rationale for the addition of SCID screening to NBS programs of other states.
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