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Research Studies is a monthly compilation of research articles funded by AHRQ or authored by AHRQ researchers and recently published in journals or newsletters.
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Chen HF, Rose AM, Waisbren S
Newborn screening and treatment of phenylketonuria: projected health outcomes and cost-effectiveness.
This study’s objective was to evaluate the cost-effectiveness of newborn screening and treatment for phenylketonuria (PKU) in the context of new data on adherence to a recommended diet treatment and a newly available drug (sapropterin dihydrochloride). A computer simulation model was developed to project outcomes for a hypothetical cohort of newborns which compared four strategies. The strategies compared were: 1) clinical identification (CI) with diet treatment; 2) newborn screening (NBS) with diet treatment; 3) CI with diet and medication; and 4) NBS with diet and medication. The range of incremental cost-effectiveness ratio went from $6400/QALY for newborn screening with diet treatment compared to clinical identification with diet treatment up to $16,000,000/QALY for adding medication to NBS with diet treatment. Future research is needed to consider conditions under which sapropterin dihydrochloride would be more economically attractive.
Citation: Chen HF, Rose AM, Waisbren S . Newborn screening and treatment of phenylketonuria: projected health outcomes and cost-effectiveness. Children 2021 May 12;8(5). doi: 10.3390/children8050381..
Keywords: Newborns/Infants, Neurological Disorders, Screening, Value, Healthcare Costs