Expert Opinion on Orphan Drugs · 2016 · 11 citations · 31 references
PharmacyPediatricsPharmacoeconomicsCost-effectiveness AnalysisHealth Technology AssessmentPharmacoeconomic ModelingCost-effectiveness ComparisonCost-effectiveness ModelingEconomic EvaluationHealthcare Cost AnalysisModel-based Economic EvaluationNewborn Genetic ScreeningCost-effectivenessDiagnostic Cost AnalysisDiagnostic Test CostsHealth Economic Decision ModelingHealth Economics Of ScreeningIncremental Cost EstimationNewborn ScreeningNewborn Screening For Genetic Disorders
Objectives: This study assesses the cost-effectiveness of adding sickle-cell disease (SCD) to the Spanish newborn screening (NBS) program, and explores the sensitivity of the results to key model parameters.Methods: A discrete event simulation model was developed that compared NBS for SCD versus clinical detection. The model followed a simulated cohort of newborns for 10 years, and estimated the impact in costs and life expectancy of prophylactic treatments established after early detection. Probabilistic, one-way and two-way sensitivity analysis were performed.Results: NBS was found to be more costly and more effective than clinical detection of SCD. The estimated incremental cost per life year (LY) gained was 34,169.46 €/LY. This result was very sensitive to the cost of the screening test, the birth prevalence and the proportion of severe cases among the affected children.Conclusions: There is uncertainty regarding the cost-effectiveness of NBS for SCD in the Spanish context. Our base case estimate of the cost per LY gained lies near the 30,000€/LY commonly cited in Spain.
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Robert J. Adams, Lewis L. Hsu, Elliott Vichinsky et al. · New England Journal of Medicine · 1998 · 1.9K citations · Full text
Medicine, Hematology, Pediatrics +10
Causes and Outcomes of the Acute Chest Syndrome in Sickle Cell Disease
Elliott Vichinsky, Lynne Neumayr, Ann N. Earles et al. · New England Journal of Medicine · 2000 · 1.2K citations · Full text
Medicine, Cardiology, Hematology +7
Effect of Hydroxyurea on Mortality and Morbidity in Adult Sickle Cell Anemia
Martin H. Steinberg, Franca B. Barton, Oswaldo Castro et al. · JAMA · 2003 · 899 citations · Full text