Cost-effectiveness analysis of newborn screening for sickle-cell disease in Spain. (2nd June 2016)

• Record Type: Journal Article
• Authors: Castilla-Rodríguez, Iván; Cela, Elena; Vallejo-Torres, Laura; Valcárcel-Nazco, Cristina; Dulín, Elena; Espada, Mercedes; Rausell, Dolores; Mar, Javier; Serrano-Aguilar, Pedro
• Is Part Of: Expert opinion on orphan drugs. Volume 4:Number 6(2016:Jun.)
• Page Start: 567
• Page End: 575
• Publication Date: 2016-06-02
• Subjects: Cost-effectiveness -- discrete-event simulation -- newborn screening -- sickle-cell disease; Orphan drugs -- Periodicals; Rare diseases -- Periodicals; Chemotherapy -- Periodicals; 615.1
• DOI: 10.1080/21678707.2016.1179572
• Languages: English
• ISSNs: 2167-8707

Abstract

ABSTRACT: 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.