FDA Expands RWE Demonstration Project

Regulatory NewsRegulatory News | 10 April 2019 |  By 

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 already completed RCTs, and now the project also seeks to predict the results of the seven additional RCTs that are still ongoing. The overall program aims to identify when and where RWE based on health care databases can provide 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.

“Predicting the results of clinical trials before they’re completed is important for showing that in certain circumstances real-world evidence could potentially substitute for a trial,” said Jessica Franklin, assistant professor of medicine at Harvard Medical School, biostatistician at Brigham and Women’s Hospital, and lead of RCT DUPLICATE. “This additional work will be vital as we develop a process model for the implementation of regulatory-grade RWE studies.”

RCT DUPLICATE researchers are running all analytics on the Aetion Evidence Platform, which is backed by investors including McKesson Ventures, Sanofi Ventures, Amgen Ventures and UCB. Aetion is also training FDA staff on its platform to enable them to review a completed RWE study and test its robustness.

"Aetion is training FDA in small teams in order to mimic a review of an RWE submission. The training will allow FDA to understand how parameters can be modified in the Aetion Evidence Platform to test a study’s robustness and ultimately increase confidence in the results," a company spokesperson said.

RCT DUPLICATE anticipates interim results in mid-2019 and full results at the end of 2020. The researchers have already registered an initial pilot study, which will inform the forthcoming seven studies.

In addition, through a grant funded by the National Institutes of Health, RCT DUPLICATE researchers are conducting RWD analyses on more than 25 cardiovascular studies ongoing Phase IV trials and published Phase III/IV trials. 


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