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Health and economic outcomes of newborn screening for infantile-onset Pompe disease
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4-2021
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Source: Genet Med. 23(4):758-766
[PDF-687.44 KB]
Details:
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Alternative Title:Genet Med
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Description:PURPOSE:
To estimate health and economic outcomes associated with newborn screening (NBS) for infantile-onset Pompe disease in the United States.
METHODS:
A decision analytic microsimulation model simulated health and economic outcomes of a birth cohort of 4 million children in the United States. Universal NBS and treatment was compared with clinical identification and treatment of infantile-onset Pompe disease. Main outcomes were projected cases identified, costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) over the life course.
RESULTS:
Universal NBS for Pompe disease and confirmatory testing was estimated to cost an additional $26 million annually. Additional medication costs associated with earlier treatment initiation were $181 million; however, $8 million in medical care costs for other services were averted due to delayed disease progression. Infants with screened and treated infantile-onset Pompe disease experienced an average lifetime increase of 11.66 QALYs compared with clinical detection. The ICER was $379,000/QALY from a societal perspective and $408,000/QALY from the health-care perspective. Results were sensitive to the cost of enzyme replacement therapy.
CONCLUSION:
Newborn screening for Pompe disease results in substantial health gains for individuals with infantile-onset Pompe disease, but with additional costs.
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Pubmed ID:33281187
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Pubmed Central ID:PMC8035228
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