Developing a comprehensive framework for Real-World Evidence in regulatory decisions: global lessons with...

The integration of Real-World Evidence (RWE) into regulatory frameworks has gained global interest to complement traditional clinical data, particularly where randomized controlled trials may be limited. RWE refers to clinical evidence derived from the analysis of Real-World Data (RWD), including information routinely collected from sources such as electronic health records, claims databases and registries. The increasing reliance on large-scale digital data also raises challenges related to privacy, inclusivity and equity, highlighting the need for transparent and socially responsible approaches. This study developed a consensus-based framework for incorporating RWE into regulatory decision-making in Colombia, with potential applications in similar Global South contexts.

A two-phase design was applied. Phase I was a scoping review following Joanna Briggs Institute guidelines to identify RWE frameworks and guidelines. Searches in databases and regulatory agencies yielded 61 relevant documents. In Phase II, an expert panel of ten professionals in RWE, RWD, and regulation refined preliminary recommendations through a modified Delphi approach, reaching consensus on their applicability.

The scoping review revealed a high concentration of RWE studies originating from the United States and Europe, highlighting contributions by agencies such as the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in guiding RWE for regulatory decisions, particularly in oncology and rare diseases. The expert panel validated 25 recommendations, grouped into six clusters addressing selection, transparency, stakeholder engagement, analytical validation, accessibility and capacity.

This framework guides Colombia and the Global South in strengthening evidence-based regulation. It emphasizes governance, transparency, inclusivity and privacy safeguards, supporting equitable decision-making aligned with international standards.