Il ruolo della Real World Evidence nella fase pre-marketing dei farmaci: le esperienze e le prospettive future di Fondazione ReS nel delineare i confini delle Target Population.

The role of Real-World Evidence (RWE) concerns the entire drug lifecycle; despite widely recognised in the post-marketing, it is still debated in the pre-marketing, mainly as per the identification and analysis of target populations (TPs) for new drugs or indications. Through administrative healthcare databases, Research and Health Foundation (ReS), in collaboration with experts, develops algorithms to select and analyse TPs. As of March 2024, 85 TPs in 15 clinical areas have been analysed, of which oncology is the most represented. Findings on prevalence and incidence of specific diseases (or subpopulations), patient characteristics and costs directly charged to the Italian National Health Service, are provided. These are useful for healthcare institutions and pharmaceutical companies. In the future, efforts will focus on the development of tools based on artificial intelligence and synthetic data to improve analyses of TPs and support regulatory decisions on drugs.