Abstract

Clinical trial diversity has become an increasingly important focus in medicine development, as clinical evidence should reflect the populations who will receive these medicines. Historical examples have demonstrated that limitations in population representativeness may influence dosing recommendations, safety assessments, labelling and regulatory decision-making long after marketing authorisation.

Through selected case studies, this article explores how evidence gaps related to sex, ethnicity and representative trial populations have shaped regulatory outcomes across different therapeutic areas. It also examines how evolving regulatory expectations, health technology assessment (HTA) and emerging regulatory science, including real-world evidence (RWE), model-informed drug development (MIDD), physiologically-based pharmacokinetic (PBPK) modelling and population pharmacokinetic (PopPK) modelling, are contributing to more robust evidence generation throughout the development process.

Finally, the article discusses the evolving role of regulatory affairs in anticipating evidence gaps and integrating complementary evidence-generation approaches to support more informed regulatory and healthcare decision-making.