OFF-LABEL MEDICINES AND THE RIGHT TO HEALTH: REGULATORY CHALLENGES, LEGAL IMPLICATIONS AND COMPARISON BETWEEN BRAZIL, THE UNITED STATES AND THE EUROPEAN UNION.

Abstract

The use of off-label medications, defined as the prescription of 
drugs for purposes not specified in the label approved by 
regulatory agencies, is a growing practice in contemporary 
medicine. This phenomenon is often linked to the lack of 
alternative therapies or the existence of favorable scientific 
evidence; however, it raises concerns regarding safety and 
efficacy. The central issue lies in the judicialization of healthcare, 
where patients seek access to off-label medications through legal 
action, burdening the healthcare system and creating complex 
legal precedents. This study aims to analyze the regulation, 
challenges, and legal implications of off-label drug use in Brazil, 
with a comparative analysis between Brazil, the United States, 
and the European Union. The methodology includes a literature 
review and documentary analysis of legislation, case law, and 
scientific studies on the topic. The results indicate that the lack of 
clear regulation in Brazil contributes to inconsistent and unsafe 
practices, in addition to intensifying judicialization. In contrast, the 
United States and the European Union adopt distinct but equally 
rigorous approaches to the regulation of off-label use. It is 
concluded that more robust regulation and the establishment of 
strict criteria for off-label prescriptions may reduce risks and 
mitigate the negative impacts of judicialization in Brazil, 
promoting greater safety for patients and healthcare 
professionals. The differences between the regulatory frameworks of the Brazilian Health Regulatory Agency 
(ANVISA), the United States Food and Drug Administration 
(FDA), and the European Medicines Agency (EMA) highlight the 
need for ongoing debate on the role of regulatory agencies, 
medical professional autonomy, and patient protection, 
especially in the face of increasing judicialization of healthcare.