USA – FDA Expands RWE Demonstration Project

The US Food & Drug Administration (FDA) and Brigham and Women’s Hospital on Wednesday announced they are expanding a demonstration project using real-world evidence (RWE) to predict the results of seven ongoing Phase IV trials. The expanded program means researchers for the first time will estimate the results of randomized controlled trials (RCTs) that have not yet concluded.

Funded by FDA’s Center for Drug Evaluation and Research and led by FDA’s Office of Medical Policy, the project known as RCT DUPLICATE seeks to replicate select RCTs using claims databases as part of an effort to figure out which clinical effectiveness questions can be answered with real-world data (RWD).

“Such findings will lay the groundwork for regulators to gain confidence in their decision making of whether an RCT can be substituted with a RWD study,” the researchers said. “Our goal is to understand for what types of clinical questions real world data analyses can be conducted with confidence and with which designs and analysis methods.”

Originally, the demonstration project sought to replicate the results of 30 RCTs, but now the project seeks to identify when and where RWE based on health care databases can provide robust estimates of treatment safety and efficacy for supplemental new drug applications.

The demonstration project is part of the FDA’s RWE Program, as mandated by the 21st Century Cures Act