Original Title: Coste-efectividad del cribado neonatal de la deficiencia primaria de carnitina

Authors' 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.

Authors' results and conclusions: 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%.

Authors' methods: 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.

Project Status: Completed

Year Published: 2014

URL for published report: https://sescs.es/coste-efectividad-del-cribado-neonatal-de-la-deficiencia-primaria-de-carnitina/

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: Spain

Organisation Name: Canary Health Service

Contact Address: Dirección del Servicio. Servicio Canario de la Salud, Camino Candelaria 44, 1ª planta, 38109 El Rosario, Santa Cruz de Tenerife

Contact Name: sescs@sescs.es

Contact Email: sescs@sescs.es