2013

2013, ETS CN, Informes

Cost-effectiveness of the newborn screening of biotinidase deficiency

Full title: Cost-effectiveness of the newborn screening of biotinidase deficiency

Authors: Vallejo Torres L, Castilla Rodríguez I, Cuéllar Pompa L, Couce Pico ML, Pérez Cerdá C, Martín Hernández E, Pineda Marfa M, Campistol Plana J, 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 three additional conditions, including biotinidase deficiency. 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.

Aims

To evaluate the cost-effectiveness of incorporating biotinidase deficiency to the series of conditions being screened in the newborn screening programme in Spain, and to determine the budget impact of its implementation.

Methods

We developed a cost-effectiveness model that compared two options: including biotinidase deficiency to the diseases screened in the national screening programme and to not include this condition in the early detection programme and diagnose the condition by clinical diagnosis. The model takes into account 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 and the Health Care System for the Autonomy and Care of Dependent Persons in Spain, expressing the costs in 2013 prices. We estimated the costs 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 Years (QALY), which combine information on the quality of life and length of life of the individuals. 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 biotinidase deficiency is both cheaper and more effective. When taking the long term perspective, the cost-savings related to the programme per child are 1.4 € and 2.4 €, when the cost of the screening test is 1.9 € and 0.9 €, respectively. The results for the budget impact, which considers the incremental cost per year of the programme, shows that the cost-savings comes from treatment costs avoided in the long run. The programme would generate cost-savings from the 6th or from the 15th year, depending on the price of the test, 1.9 € and 0.9 €, respectively. The results were found to be robust in the sensitivity analysis, estimating that newborn screening for biotinidase deficiency is cost-effective with a probability of 94% and for a willingness to pay per QALY gain of 30,000 €/QALY.

Conclusions

Newborn screening for biotinidase deficiency was found to be cost-effective, leading to both a clinical improvement and a reduction in the long term costs when considering the benefits of early detection in the long run.

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2013, ETS CN, Informes

Cost-effectiveness of the newborn screening of classic galactosemia

Full title: Cost-effectiveness of the newborn screening of classic galactosemia

Authors: García Pérez L, Valcárcel Nazco C, Castilla Rodríguez I, Vallejo Torres L, Briones Godino P, Ruíz Pons M, Vitoria Miñana I, Cuéllar Pompa L, Serrano Aguilar P.

Contact person: Lidia García Pérez (lidia.garciaperez@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 classic galactosemia. 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 a newborn screening programme for classic galactosemia in Spain, comparing health results and costs of the screening with the clinical diagnosis.

Method

We developed a cost-effectiveness model that compared two options: including classic galactosemia to the national screening programme and to not include this condition in the early detection programme and diagnose the condition by clinical diagnosis. The model takes into account the life expectancy of the newborns. The perspective of the analysis was that of the National Health Service and the Health Care System for the Autonomy and Care of Dependent Persons in Spain, expressing the costs in 2013 prices. We estimated the costs related to the screening programme including screening tests, confirmation tests, as well as treatment and follow up costs of those detected by the programme. Two alternative types of tests screening, MS/MS and fluorometric techniques. 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 Life Years gained (LY). Health outcomes and costs were discounted at a 3% rate. We undertook deterministic analyses and a probabilistic sensitivity analysis using Monte Carlo simulation.

Results

According to the results of this study, newborn screening for classic galactosemia is not cost-effective. For a lifetime horizon, the incremental costs per newborn are 6.49 €, and the incremental LYs per newborn are 0.000050135, giving an incremental cost effectiveness ratio of 129,464 €/LY. The sensitivity analysis shows the effect of the uncertainty on the results of the model. The sensitivity analysis estimated that the probability of a cost-effective classic galactosemia screening programme was inferior to 10% for a willingness to pay of 30,000 €/LY.

Conclusions

The introduction of neonatal screening for classic galactosemia is not cost effective for a willingness to pay of 30,000 €/LY.

PDF (in spanish)

2013, ETS CN, Informes

Cost-effectiveness of the newborn screening of congenital adrenal hyperplasia

Full title: Cost-effectiveness of the newborn screening of congenital adrenal hyperplasia

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

Contact person: Iván Castilla Rodríguez (ivan.castillarodriguez@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 three additional conditions, including congenital adrenal hyperplasia. 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 incorporating congenital adrenal hyperplasia to the series of conditions being screened in the newborn 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 congenital adrenal hyperplasia 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 life expectancy, by taking into account deaths avoided by screening, and expressed as life-years gained (LYG). Health outcomes and costs were discounted at a 3% rate. We undertook a one-way sensitivity analysis on those parameters with the highest impact on the results and those surrounded by the highest uncertainty, and a probabilistic sensitivity analysis using Monte Carlo simulation.

Results

According to the results of this study, newborn screening for congenital adrenal hyperplasia is more costly and more effective than clinical detection. For a lifetime horizon, the incremental costs per newborn are € 1.71, and the incremental life years per newborn are 0.00022, giving an incremental cost effectiveness ratio of € 7,899/LYG, which is below € 30,000/LYG, i.e. the usual threshold used in Spain. The sensitivity analysis estimated that newborn screening for congenital adrenal hyperplasia is cost-effective with a probability of 88.5% and for a willingness to pay per life year gain of € 30,000/LYG. Nevertheless, there is a high uncertainty surrounding the sensitivity of the clinical detection (especially in the salt-wasting form).

Conclusions

According to the available evidence when developing this report, the introduction of neonatal screening for congenital adrenal hyperplasia would be cost effective for a willingness to pay € 30,000/LYG, assuming a clinical sensitivity for the salt-wasting form of 85% when screening is not available. Nevertheless, these results should be considered carefully due to the high uncertainty surrounding the estimation of the clinical sensitivity.

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