Cost-effectiveness of the newborn screening of cystic fibrosis in Spain

Full title: Cost-effectiveness of the newborn screening of cystic fibrosis in Spain

Authors: Valcárcel-Nazco C, Oliva Hernández C, Velasco González V, Cuéllar-Pompa L, Castilla I, Vallejo-Torres L, Serrano-Aguilar P.

Contact person: Cristina Valcárcel Nazco (cristina.valcarcelnazco@sescs.es)

Summary

Introduction

CF is the autosomal recessive genetic lethal disease most common between white people. It affects many vital organs, being lung disease the responsible for most of deaths. Its incidence in Spain is estimated between 1/6.496 and 1/4.500 newborns per year so CF is included among rare disease.

During the last two decades each Spanish autonomous community has decided the inclusion of newborn screening programs, such as CF neonatal screening, requiring neither prior technology assessment reports nor consensus within the NHS.

Objectives

To determine the cost-effectiveness ratio of each of the main strategies for neonatal screening for cystic fibrosis in Spain.

Method

We have performed a full economic evaluation that compares the cost-effectiveness ratio of each of the main CF newborn screening strategies that are currently being carried out in Spain: IRT/IRT, IRT/DNA, IRT/IRT/DNA and IRT/DNA/IRT versus no screening. We have developed a decision tree with five main branches representing each strategy to be evaluated and the option of not screening the disease. The measures of effectiveness used were LYGs and QALYs. Both cost and effects were discounted at a rate of 3%. A WTP of € 30,000/LYG and € 30,000/QALY was considered. Costs were expressed in euros of 2013.

A probabilistic sensitivity analysis was performed by means of Monte Carlo simulations, allowing us to compute the acceptability curves and the expected value of perfect information.

To determine the parameters that caused greater variability in the model results, ANCOVA models were applied to Monte Carlo simulations.

Results

Results of the economic evaluation model show that newborn screening for CF is an efficient alternative. The IRT+IRT+DNA screening strategy has a lower ICER compared with no screening (8,801.46 €/LYG and 4,024.85 €/QALY). The screening strategy with higher ICER is IRT+DNA+IRT (16,394.59 €/LYG y 7,497.14 €/QALY).

Conclusions

CF neonatal screening is an efficient alternative in terms of cost-effectiveness from the perspective of the NHS, resulting in a profit on health outcomes of patients in both LYG and QALY.