OBJECTIVES: The Brazilian Constitution establishes that the State must ensure integral, universal and free health care. The financing of this model is impacted by judicial decisions that require the supply of medicines, in spite of the guidelines and lists. Knowledge of the impact of these court decisions is essential to the development of strategies that ensure the provision of evidence-based treatment and protect the sustainability of the system. Legal decisions determine that the Ministry of Health (MH) treat patients outside established guidelines. Miglustate is available in the Brazilian public health system (SUS) to treat Gaucher disease. This paper aims to measure the relevance and impact of judicialization on miglustate distributions in 2019.

METHODS: From MH administrative information, was identified the number of miglustate that was sent to patients who were in accordance with official (“regular”) guidelines and for who weren’t in accordance with official (“judicial order”) guidelines were identified in 2019. Cost and quantity of miglustate sent to each group were established and the exchange rate of US$1: BRL 3.30.

RESULTS: In the evaluation period, 46 patients used miglustate distributed by the MH, being 25 (54%) "regular" and 21 (46%) "judicial". These patients received 18,750 tablets and 13,770 miglustate tablets, respectively, bringing a total cost of $ 849,514/year. Judicial order accounted for 38% of total spending and treated patients with: Niemann Pick C Disease (71%), Tay-Sachs Syndrome (19%), and Gangliosidosis (10%). In Brazil, the use of miglustate for Tay-Sachs Syndrome and Gangliosidosis is not approved, characterized as off-label use.

CONCLUSIONS: Court decisions accounted 38% of MH spending on miglustate in 2019 and subject patients to treatment without systematic guidance in official protocols. Using miglustate outside official indications endangers patient safety and has a significant budgetary impact on the SUS. The technical qualification of the judiciary is required from the perspective public and evidence-based medicine.