Custo-efetividade da incorporação do Espectrômetro de Massa em Tandem no Programa Nacional de Triagem Neonatal para diagnóstico da Fenilcetonúria e da MCAD.

Abstract

The Brazilian Neonatal Screening National Program screens Phenylketonuria (PKU), Congenital Hypothyroidism, Cystic Fibrosis, Hemoglobinopathies and Disability Biotinidase. The National Commission for Incorporation for Technology Incorporation recently recommended the acquisition of the Mass Spectometer in Tandem (MS/MS) to diagnose rare diseases. The application of this new technology can expand the detection of Inborn Errors of Metabolism. In this way we sought to compare different scenarios of neonatal screening (NS) with the currently technology (Fluorimetry) used only for PKU, and MS/MS screening for PKU and Disability Medium Chain Acyl-Coenzyme Dehydrogenase (MCAD). Therefore it designed a mathematical model based on probabilistic Markov chains that simulated NS of PKU and MCAD as well as the natural history of MCAD (no screening). It was simulated with a hypothetical cohort of 2 million newborn. The time horizon adopted was life expectancy of the population -78 years according to Brazilian Institute of Geography and Statistics. A sensitivity analysis probabilistic based on Monte Carlo simulation was performed in order to build an acceptability threshold for incorporating the technology in question. It was used a 5% discount rate for costs and clinical consequences. The study conclusion points the scenario of MS/MS more cost effective for NS of PKU and MCAD when compared to the current scenario (Fluorimetry to screen PKU and no screening of MCAD). It provides a gain of 14,57 QALY and reduces the cost of TN in 29%, that represents a resource saving of R$ 708 million per year. The scenario of the conventional screening for PKU did not have a substantial gain in effectivity (0,562 QALY), and also more onerous (R$ 2,5 billion) to the Public Health System.