Newborn Screening and Treatment of Phenylketonuria: Projected Health Outcomes and Cost-Effectiveness.

ABSTRACT: The objective of this study was to evaluate the cost-effectiveness of newborn screening and treatment for phenylketonuria (PKU) in the context of new data on adherence to recommended diet treatment and a newly available drug treatment (sapropterin dihydrochloride). A computer simulation model was developed to project outcomes for a hypothetical cohort of newborns with PKU. Four strategies were compared: (1) clinical identification (CI) with diet treatment; (2) newborn screening (NBS) with diet treatment; (3) CI with diet and medication (sapropterin dihydrochloride); and (4) NBS with diet and medication. Data sources included published literature, primary data, and expert opinion. From a societal perspective, newborn screening with diet treatment had an incremental cost-effectiveness ratio

SUBMITTER: Chen HF 

PROVIDER: S-EPMC8151371 | biostudies-literature | 2021 May

REPOSITORIES: biostudies-literature