https://pubmed.ncbi.nlm.nih.gov/38093484
Title: A Comparison of Real-World Data and Real-World Evidence for Regulatory Decision-Making: A Review of FDA and EMA Publications
Real-world data (RWD) and real-world evidence (RWE) are becoming increasingly important in regulatory decision-making for drug approvals. However, regulatory agencies and stakeholders may have different definitions for RWD and use different criteria to determine when analysis of such data is considered RWE.
In a recent study, researchers from the US Food and Drug Administration (FDA) and the Centers for Medicare & Medicaid Services reviewed two prominent publications describing the use of RWE in drug approvals by the FDA and the European Medicines Agency (EMA). The review aimed to explore the inconsistencies and opportunities for developing a consistent understanding of the role of RWE in regulatory decision-making.
Both publications considered non-interventional (observational) studies, RWD as a comparator arm for a single-arm trial, product-related literature reviews, and RWD to support clinical trial implementation (e.g., to identify potential participants) as generating RWE. However, there were inconsistencies regarding the types of data sources and study designs that were considered as not generating RWE.
For example, there was no agreement regarding whether RWE is generated when RWD describe therapeutic contexts or are used in phase I/II interventional trials, open-label extension studies, or pharmacovigilance activities.
The findings highlight the need for a consistent understanding of the role of RWE in regulatory decision-making for drug approvals among regulatory agencies and stakeholders. The study suggests that further discussion and guidance are necessary to ensure that RWD and RWE are used appropriately and consistently in regulatory decision-making.
In conclusion, RWD and RWE play a critical role in regulatory decision-making for drug approvals. However, the lack of consistency in defining and using RWE can lead to confusion and potentially flawed decision-making. Therefore, developing a consistent understanding and guidance for the use of RWD and RWE in regulatory decision-making is crucial to ensure the safety, efficacy, and quality of approved drugs.
