Cost-effectiveness of the newborn screening of tyrosinaemia type 1

Full title: Cost-effectiveness of the newborn screening of tyrosinaemia type 1

Authors: Vallejo-Torres L, Castilla I, Dulín-Íñiguez E, Espada M, Rausell D, Valcárcel-Nazco C, Cuéllar-Pompa L, Serrano-Aguilar P.

Contact person: Laura Vallejo Torres (laura.vallejotorres@sescs.es)

Summary

Introduction

The Ministry of Health, Social Services and Equality aims to reduce the inequalities in the newborn screening programmes offered in the different regions in Spain. After the decision to increase to seven the number of conditions included in the early detection programme offered nationally, decisions makers are now considering the inclusion of additional conditions, including tyrosinaemia type 1. Newborn screening programmes have to meet a series of requirements, including providing evidence on the cost-effectiveness of the programme, by comparing the costs and health outcomes of early detection to those related to the clinical diagnosis.

Objectives

To evaluate the cost-effectiveness of an early detection programme for tyrosinaemia type 1 in the framework of thenewborn screening programme in Spain, and to determine the budget impact of its implementation.

Method

We developed a cost-effectiveness model that compared two options: including tyrosinaemia type 1 to the diseases screened in the national screening programme and to no include this condition in the early detection programme and diagnose the condition by clinical diagnosis. The model takes into account to the life expectancy of the newborns, capturing the impact on the long term of the early detection. The perspective of the analysis was that of the National Health Service in Spain, expressing the costs in 2013 prices. We estimated the cost related to the screening programme including screening tests, confirmation tests, as well as treatment and follow up costs of those detected by the programme. These costs were compared with those related to the clinical diagnosis of the condition. The differences in the costs were then compared with the difference on the effectiveness. Effectiveness was measured using Quality Adjusted Life Year (QALYs). Health outcomes and costs were discounted at a 3% rate. We undertook a probabilistic sensitivity analysis using Monte Carlo simulation.

Results

According to the results of this study, newborn screening for tyrosinaemia type 1 is more costly and more effective than clinical detection. For a lifetime horizon, the incremental costs per newborn are 1.13 €, and the incremental QALY gain per newborn is 0.00003, yieldind to an incremental cost effectiveness ratio of 40,670 €/QALY. The sensitivity analysis, shows that the probability that newborn screening for tyrosinaemia type 1 is cost-effective is of 24% for a willingness to pay of 30,000 €/QALY. The budget impact analysis shows that the initial additional cost per child of the newborn screening programme of tyrosinaemia type 1 is low, around 0.44 € per newborn.

Conclusions

This study estimates that the incremental cost of the introduction of neonatal screening for tyrosinaemia type 1 is around 40,000€/QALY, which is above the reference willingness to pay of 30,000 €/QALY commonly used in these types of studies in Spain. For willingness to pay higher than 40,000 €/QALY, the inclusion of tyrosinaemia type 1 in the newborn screening programme would be considered cost-effective. The budget impact analysis shows that the incremental initial cost is relatively low.