Scientific production on the regulation of unregistered medicines in Brazil, the United States, and Europe

Vigil Sanit Debate, Rio de Janeiro, 2025, v.13: e02285 | Published on: 17/01/2025

Abstract

Introduction: The use of unregistered drugs indicates a shift in the regulatory paradigm for innovative medicines by shortening access to drugs that are still in the experimental phase, which also increases uncertainties regarding the risks associated with their use. Objective: to identify the characteristics of scientific publications on the regulation of unregistered drugs in Brazil, the United States, and Europe. Method: This is a descriptive, quantitative study involving bibliometric analysis. A total of 58 studies were selected based on the descriptors and eligibility criteria from the SciELO, Portal Capes, and Web of Science databases. Absolute and relative frequency analyses, as well as averages of the categories listed in the analysis plan, and keyword profiles using VOSviewer® were performed. Results: Researchers’ interest in this topic has been growing since 2016. Academic production was concentrated in the United States, with original articles having low methodological rigor, and focused on medical journals. Some studies declared conflicts of interest with the pharmaceutical industry. The central themes were  “aspects of the regulatory framework for expanded access/compassionate use” and “ethics in the use of unregistered drugs,” with the main keywords being: expanded access, efficacy, safety, Covid-19, risk, emergency authorization, and real-world data.  Conclusions: The regulation of unregistered drugs deserves further exploration through high-quality studies in the field of Public Health, particularly in  countries of the global south, with research that focuses on building elements of individual and collective health protection in the regulation of unregistered drugs.