Health and economic outcomes of newborn screening for infantile-onset Pompe disease.

ABSTRACT:

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

SUBMITTER: Richardson JS 

PROVIDER: S-EPMC8035228 | biostudies-literature | 2021 Apr

REPOSITORIES: biostudies-literature