Análise e gestão de riscos raros em saúde: um estudo de caso do medicamento zolgensma

Abstract

Spinal Muscular Atrophy (SMA) is a rare genetic disease characterized by muscular atrophy secondary to the degeneration of motor neurons located in the anterior horn of the spinal cord. In 2023, the National Supplementary Health Agency (ANS) included the drug Zolgensma in the list of minimum procedures to be provided by Health Plan Operators (HPO), considered the most expensive drug in the world, costing more than 6 million Brazilian Reais. This study aimed to conduct a Monte Carlo simulation (MCS) based on the profile of beneficiaries of a large HPO (with over 100,000 beneficiaries) to understand the financial impact of making the drug available. For the medication to be provided, the beneficiary must be a maximum of 6 months old and have SMA type I. As a result of the MCS, up to 19 occurrences of medication requests were observed, with a maximum cost of R$128,932,078.72. It is emphasized the need to redo the MCS with new rare risk events identified by the HPO.