The use of drugs for clinical conditions that differ from those approved in prescribing information (product labels) is known as off-label use. In Brazil, the Brazilian Health Regulatory Agency (ANVISA) is the official organization that regulates the use of health technologies. For technologies to be incorporated into the Brazilian public health system, registration with ANVISA is mandatory. However, occasionally, it is necessary to evaluate technologies for off-label use in the interests of public health. This study aimed to identify the health technologies recommended by the National Committee for Health Technology Incorporation (CONITEC) with an off-label indication between January 2012 and October 2017.

A descriptive study was undertaken using data available on the CONITEC website.

The study identified seven drugs with a favorable recommendation for off-label use: everolimus, sirolimus, and tacrolimus as immunosuppressants in transplant recipients; clozapine for bipolar affective disorder; pentoxifylline for cutaneous leishmaniosis mucosa; risperidone for adults with autism spectrum disorder, and bevacizumab for age-related macular degeneration and diabetic macular edema. For these decisions the Committee considered the scientific evidence available for the indication proposed, the severity of the disease, and the existence or absence of alternative treatments. This was possible because Brazilian legislation allows ANVISA to authorize the off-label use of health technologies provided that the analysis is supported by scientific evidence regarding effectiveness, accuracy, and safety for the intended purpose.

The off-label use of health technologies is a worldwide practice that can favor vulnerable populations and neglected diseases. This practice should be seen as positive when there is evidence supporting off-label use, and such decisions should not be influenced by political, economic, or marketing considerations.