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 of $6400/QALY compared to clinical identification with diet treatment. Adding medication to NBS with diet treatment resulted in an incremental cost-effectiveness ratio of more than $16,000,000/QALY. Uncertainty analyses did not substantially alter the cost-effectiveness results. Newborn screening for PKU with diet treatment yields a cost-effectiveness ratio lower than many other recommended childhood prevention programs even if adherence is lower than previously assumed. Adding medication yields cost-effectiveness results unlikely to be considered favorable. Future research should consider conditions under which sapropterin dihydrochloride would be more economically attractive.

Objectives. There are several approaches such as presumed consent and compensation for deceased donor organs that could reduce the gap between supply and demand for kidneys. Our objective is to evaluate the magnitude of the economic impact of policies to increase deceased donor organ donation in the United States. Methods. We built a Markov model and simulate an open cohort of end-stage renal disease patients awaiting kidney transplantation in the United States over 20 years. Model inputs were derived from the United States Renal Data System and published literature. We evaluate the magnitude of the health and economic impact of policies to increase deceased donor kidney donation in the United States. Results. Increasing deceased kidney donation by 5% would save $4.7 billion, and gain 30,870 quality-adjusted life years over the lifetime of an open cohort of patients on dialysis on the waitlist for kidney transplantation. With an increase in donations of 25%, the cost saved was $21 billion, and 145,136 quality-adjusted life years were gained. Policies increasing deceased kidney donation by 5% could pay donor estates $8000 or incur a onetime cost of up to $4 billion and still be cost-saving. Conclusions. Increasing deceased kidney donation could significantly impact national spending and health for end-stage renal disease patients.

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