OBJECTIVES : Despite the increasing use of Real-World Data (RWD) and Real World Evidence (RWE) as supportive evidence for safety and efficacy assessments, the absence of specific guidelines and regulations for RWE generation is one of the main hurdles for its use in regulatory decision making in Latin America (LATAM). In order to assist LATAM regulatory agencies on the utilization and appraisal of RWE, we aimed to explore desirable evidence features for regulatory decision making.

METHODS : We conducted a targeted review of case studies and other available documentation in European and North American countries to understand the main study designs and evidence requirements for regulatory utilization.

RESULTS : Usage of RWD/RWE for new drug approvals and label extension was reported in the FDA and EMA from 2017 onwards. Moreover, some drugs like Blinatumomab and Cerliponase alfa, approved with RWD/RWE by both agencies, were also approved in LATAM. Evidence appropriateness and reliability were identified as key aspects to check when collecting RWD for regulatory purposes. According to the FDA RWE framework, the utilization of common data models (e.g. OMOP, Sentinel, PCORnet) allows for data integrity and conformity verification, whereas existent guidelines enable the critical appraisal of population selection and data plausibility. Finally, RWE generation initiatives were identified across the region (from nationwide health information systems, to disease registries and institutional EMR analyses), but none of these sources have been adequately assessed for its use in regulatory decision support.

CONCLUSIONS : RWD/RWE quality checks and standards have already been developed and could be used by local agencies to aid regulatory decisions involving Real World Studies and to develop local or reginal guidelines.