Informes

2014, ETS CN, Informes

Cost-effectiveness of the newborn screening of primary carnitine deficiency

Full title: Cost-effectiveness of the newborn screening of primary carnitine deficiency

Authors: Castilla I, Vallejo-Torres L, Couce ML, Del Toro M, Gorostiza I, García-Hernández L, Dulín-Íñiguez E, Espada M, Valcárcel-Nazco C, García-Pérez L, Posada M, 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 primary carnitine 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.

Objectives

To evaluate the cost-effectiveness of incorporating primary carnitine deficiency 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: to include primary carnitine deficiency to the diseases screened in the national screening programme, and to diagnose this 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 2014 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 in life years gained. Health outcomes and costs were discounted at a 3% rate. We undertook a probabilistic sensitivity analysis using Monte Carlo simulation. Finally, we analysed the budget impact of including newborn screening for primary carnitine deficiency using a five-years’ time horizon.

Results

According to the results of this study, newborn screening for primary carnitine deficiency is more costly but more effective than clinical detection. For a lifetime horizon and the estimated amount of neonates who born in Spain annually, around 450,000, the incremental costs are 249,076 € (0.55 € per newborn), and the incremental LYs are 17.52 (0.00004 per newborn), giving an incremental cost effectiveness ratio of 14,217.56 €/LY. The results were found to be sensitive to the probability of sudden infant death among those cases that remain undetected. However, the probabilistic sensitivity analysis estimated that newborn screening for primary carnitine deficiency is cost-effective with a probability of 73.9% and for a willingness to pay of 30,000 €/LY. The budget impact for the Spanish newborn children was 49,153 € first year and 72,459 € fifth year.

Conclusions

The introduction of neonatal screening for primary carnitine deficiency is cost effective for a willingness to pay 30,000 €/LY, and the probability of being cost-effective is around 73.9%.

PDF (in spanish)

2014, ETS CN, Informes

Cost-effectiveness of the newborn screening of long-chain acyl-CoA dehydrogenase deficiency

Full title: Cost-effectiveness of the newborn screening of long-chain acyl-CoA dehydrogenase deficiency

Authors: Castilla I, Vallejo-Torres L, Couce ML, Gorostiza I, García-Hernández L, Dulín-Íñiguez E, Espada M, Valcárcel-Nazco C, García-Pérez L, Posada M, 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 very long-chain acyl-CoA dehydrogenase deficiency (VLCADD). 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 VLCADD 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: to include VLCADD to the diseases screened in the national screening programme, and to diagnose this 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 in Spain, expressing the costs in 2014 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 in life years gained. Health outcomes and costs were discounted at a 3% rate. We undertook a probabilistic sensitivity analysis using Monte Carlo simulation. Finally, we analysed the budget impact of including newborn screening for VLCADD using a five years’ time horizon.

Results

According to the results of this study, newborn screening for primary carnitine deficiency is more costly but more effective than clinical detection. For a lifetime horizon and the estimated amount of neonates who born in Spain annually, around 450,000, the incremental costs are 260,936 € (0.58 € per newborn), and the incremental LYs are 24.33 (0.000054 per newborn), giving an incremental cost effectiveness ratio of 10,723.97 €/LY. The results were found to be sensitive to the probability of sudden infant death among those cases that remain undetected, the probability of missing a case with clinical detection, and the proportion of symptomatic cases despite early detection. The probabilistic sensitivity analysis estimated that newborn screening for primary carnitine deficiency is cost-effective with a probability of 63.7% and for a willingness to pay of 30,000 €/LY. The budget impact for the Spanish newborn children was 67,956 € first year and 116,402 € fifth year.

Conclusions

The introduction of neonatal screening for VLCADD is cost effective for a willingness to pay 30,000 €/LY, and the probability of being cost-effective is around 63.7%.

PDF (in spanish)

Informes, Otros

Asenapina versus olanzapina en el tratamiento del trastorno bipolar tipo I

Título completo: Análisis coste-utilidad de la asenapina versus olanzapina en el tratamiento del trastorno bipolar tipo I

Autores (p.o. alfabético): Iván Castilla Rodríguez, Leticia Cuéllar-Pompa, Lidia García Pérez, Lilisbeth Perestelo Pérez, Pilar Pinilla Domínguez, Juan Manuel Ramos Goñi, Pedro Serrano Aguilar, Cristina Valcárcel Nazco

Persona de contacto: Juan Manuel Ramos Goñi

Objetivos

Comparar, en términos de eficiencia, la asenapina frente a la olanzapina Especialidad Farmacéutica Genérica (EFG), en el tratamiento de los  episodios  maníacos  del trastorno bipolar tipo I, tanto desde  la  perspectiva  del  Sistema  Nacional  de  Salud (SNS), como desde la perspectiva social.

ETS CN, Informes

Cost-effectiveness of the newborn screening of 3-hydroxy-3-methylglutaryl-CoA lyase deficiency (HMG)

Full title: Cost-effectiveness of the newborn screening of 3-hydroxy-3-methylglutaryl-CoA lyase deficiency (HMG)

Authors: Castilla I, Vallejo-Torres L, Linertová R, Ferrer J, Rivas Wagner E, Dulín-Íñiguez E, Valcárcel-Nazco C, García-Pérez L, Serrano-Aguilar P

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

Summary

Introduction

Homogenization of neonatal screening programs, offered in the different regions of Spain, is one of the objectives of the spanish Ministry of Health, Social Services and Equality. Following the decision to include seven diseases in the unique screening program offered by the National Health System, now a possible inclusion of 3-hydroxy-3-methylglutaryl CoA lyase deficiency (HMG) is being studied. There are several aspects to consider in the decision to incorporate new diseases in neonatal screening programs, including the study of the efficiency of the program, comparing the costs and health outcomes of early detection of disease with detection through symptomatic manifestation.

Objective

To determine the efficiency, in terms of cost-effectiveness, of the incorporation of HMG early detection in the neonatal screening program and its budgetary impact.

Methods

We have developed a cost-effectiveness model comparing two alternatives: implanting HMG neonatal screening against the clinical detection of this disease. The model follows the infants throughout their life, reflecting the impact of preventive treatments that can be set up through early detection. The perspective of the analysis was the one of National Health System, taking into account the direct costs, expressed in euro 2014. Costs relating to the screening program were estimated including the cost of screening tests, confirmatory tests and costs of treatment and monitoring of early detected patients. These costs were compared to the costs of treating HMG not detected through newborn screening. In turn, we compare the costs with the effectiveness of the screening program. The effectiveness was measured in quality-adjusted life years (QALY) gained. Both costs and effectiveness were discounted at 3%. Probabilistic sensitivity analysis was performed using Monte Carlo simulations. Finally the budgetary impact of incorporating neonatal screening of HMG for a five years time horizont was analyzed.

Results

According to the results of this economic evaluation, the neonatal screening of HMG is more expensive and there is no evidence that it is more effective than the clinical detection. Considering the number of births per year in Spain of 450,000, the increase in costs if HMG screening is implemented would be € 26,759.90 (€ 0.06 per infant). Under a number of assumptions, probabilistic sensitivity analysis estimated a 23.8% probability that the neonatal screening for this disease is cost-effective for a willingness to pay  of € 30,000 / QALY. The budgetary impact for the total annual births was estimated for Spain at € 26,290 the first year, remaining almost constant up to five years (€ 26,611).

Conclusions

With the evidence available at the time of preparation of this report we can not conclude that the introduction of HMG into the neonatal screening is cost-effective. Under a number of assumptions, and a willingness to pay at € 30,000 / QALY, the probability that the HMG screening is cost-effective is low, around 13.5%.

ETS CN, Informes

Health technology assessment reports on newborn screening

El interés internacional por los informes relacionados con cribado neonatal explica la creación de esta sección específica. Los informes completos están publicados en español y se encuentran disponibles en el apartado Ministerio de Sanidad, Servicios Sociales e Igualdad (MSSSI).

Reports 2015

Cost-effectiveness of the newborn screening of 3-hydroxy-3-methylglutaryl-CoA lyase deficiency (HMG)

Reports 2014

Cost-effectiveness of the newborn screening of long-chain acyl-CoA dehydrogenase deficiency
Cost-effectiveness of the newborn screening of primary carnitine deficiency

Reports 2013

Cost-effectiveness of the newborn screening of tyrosinaemia type 1
Cost-effectiveness of the newborn screening of congenital adrenal hyperplasia
Cost-effectiveness of the newborn screening of classic galactosemia
Cost-effectiveness of the newborn screening of biotinidase deficiency

Reports 2012

Cost-effectiveness of the newborn screening of cystic fibrosis in Spain
Cost-effectiveness of the newborn screening of sickle cell disease
Cost-effectiveness of the expanded newborn screening of congenital errors of metabolism using tandem mass spectrometry

Reports 2006

Cost-effectiveness of the newborn screening of congenital errors of metabolism using tandem mass spectrometry