Contemporary use of real-world data for clinical trial conduct in the United States: a scoping review

Abstract

Objective: Real-world data (RWD), defined as routinely collected healthcare data, can be a potential catalyst for addressing challenges faced in clinical trials. We performed a scoping review of database-specific RWD applications within clinical trial contexts, synthesizing prominent uses and themes. Materials and Methods: Querying 3 biomedical literature databases, research articles using electronic health records, administrative claims databases, or clinical registries either within a clinical trial or in tandem with methodology related to clinical trials were included. Articles were required to use at least 1 US RWD source. All abstract screening, full-text screening, and data extraction was performed by 1 reviewer. Two reviewers independently verified all decisions. Results: Of 2020 screened articles, 89 qualified: 59 articles used electronic health records, 29 used administrative claims, and 26 used registries. Our synthesis was driven by the general life cycle of a clinical trial, culminating into 3 major themes: trial process tasks (51 articles); dissemination strategies (6); and generalizability assessments (34). Despite a diverse set of diseases studied, <10% of trials using RWD for trial process tasks evaluated medications or procedures (5/51). All articles highlighted data-related challenges, such as missing values. Discussion: Database-specific RWD have been occasionally leveraged for various clinical trial tasks. We observed underuse of RWD within conducted medication or procedure trials, though it is subject to the confounder of implicit report of RWD use. Conclusion: Enhanced incorporation of RWD should be further explored for medication or procedure trials, including better understanding of how to handle related data quality issues to facilitate RWD use.