Cost-effectiveness of the newborn screening of sickle cell disease

Full title: Cost-effectiveness of the newborn screening of sickle cell disease

Authors: Castilla I, Valcárcel-Nazco C, Vallejo-Torres L, Cela E, Posada M, Dulín-Íñiguez E, Espada M, Rausell D, Mar J, Arrospide A, Serrano-Aguilar P.

Contact person: Iván Castilla Rodríguez (ivan.castillarodriguez@sescs.es)

Summary

Introduction

Neonatal screening for sickle cell disease allows the introduction of prophylactic treatments to reduce the incidence and the morbi-mortality associated with the most severe complications of this disease. However, being a rare disease, the cost-effectiveness of this secondary prevention strategy should be carefully assessed.

Objectives

To determine the efficiency of incorporating the early detection of sickle cell disease to neonatal screening programs already implemented in Spanish regions.

Method

A discrete event simulation model has been developed that compares two alternatives: implementing newborn screening for sickle cell disease in a given population that already has a neonatal screening program; or continuing with the clinical detection of this pathology. The model follows the newborns for 10 years, reflecting the impact of preventive treatments that can be established through early detection. The perspective of the analysis was that of the National Health System, taking into account the direct healthcare costs, expressed as 2013 euros. The effectiveness of the intervention was measured by using life expectancy and health related quality of life. Both the costs and effectiveness were discounted at 3%. We performed a probabilistic sensitivity analysis using 2nd order Monte Carlo simulations, which allowed the calculation of acceptability curves and the expected value of perfect information.

Results

Given the estimated birth prevalence in Spain (1:5591), there is a difference in effectiveness of 0.00005 LYGs per infant that favors HbS screening if a time horizon of 10 years is considered. By keeping costs per screened newborn at or below 1 €, neonatal screening for HbS saves costs for the same period. Screening is still efficient, for a willingness to pay of € 30,000 / LYG, if costs per screened newborn are below 2.5 €, incurring a global additional cost of 1.27 € per screened newborn, and obtaining an ICER of € 24,495.72 / LYG (€ 25,343.37 / QALY).

Conclusions

Given the estimated birth prevalence of sickle cell disease in Spain, neonatal screening of this disease is cost-effective for a time horizon of 10 years and setting a willingness to pay of € 30,000 / QALY if the cost per screened newborn does not exceed 2.5 €. The uncertainty surrounding this decision is quite high, being the probability of the screening to be cost-effective not higher than 50% in any of the scenarios assessed.