Advancing real-world evidence in Brazil: regulatory gaps and global lessons

Regulatory agencies are increasingly incorporating real-world data (RWD) and real-world evidence (RWE) into decision-making frameworks to complement randomized clinical trials. While some regions, such as the U.S. and EU, have developed structured approaches for RWE use, Brazil's regulatory environment remains comparatively limited. This study examines the status of RWE regulatory integration in Brazil through an analysis of normative documents, institutional publications, and selected case studies, using a comparative policy perspective. Although advances have been made in data standardization and the publication of technical guidelines, such as ANVISA's Guidance No. 64/2023, the practical use of RWE in regulatory processes is still nascent. Key challenges include fragmented data infrastructure, and limited intersectoral coordination. Addressing these gaps will require improved interoperability across health information systems, convergence of methodological standards, and sustained collaboration among regulatory authorities, academia, and data holders to enable consistent and scientifically robust use of RWE in the Brazilian context and, potentially, in other low- and middle-income countries.