ABSTRACT
The increasing cost of clinical drug development has led innovators to reassess the sources of data that could be useful to inform regulatory decision-making. Real-world data (RWD) can be collected from diverse sources and represents patients’ healthcare experience during routine clinical care. The refinement and analysis of data collected from RWD sources can produce real-world evidence (RWE). Currently, stakeholders are evaluating ways to apply RWD sources in current medical product development programs, including incentives for use and projects to explore novel applications. Until now, RWD has been primarily used in the post-market setting for pharmacovigilance. Moving forward, RWD could be applied in pre-market development as a suitable source for informing innovative clinical trial designs, such as platform, basket, and umbrella trials. RWD sources, such as electronic health records, claims databases, and registries could inform the selection of indications, study sites, and clinical endpoints. They may also be useful in estimating effect sizes, performing trial simulation, and providing a historical control. To collect meaningful evidence from RWD sources, bias and confounding variables must be assessed. The focus for the future will be developing novel methods for RWD collection, extraction, and analysis to modernize clinical drug development.
