For the first time, researchers from the US Food and Drug Administration (FDA) and Brigham and Women’s Hospital will estimate the results of seven ongoing trials using real-world evidence (RWE) as the two organizations expand their demonstration project.
The project – RCT Duplicate – was designed to use RWE to replicate the results of 30 completed randomized controlled trials (RCTs) to inform the FDA’s standards for using RWE in regulatory decisions.
According to Aetion, a partner in the project, the FDA’s proposal for the project was submitted through a Broad Agency Announcement and from this solicitation the regulatory agency chose Brigham and Women’s Hospital to lead the project.
A spokesperson for Aetion added, “The FDA and Brigham and Women's Hospital have a long history of collaborating and contributing to the field of real-world evidence.”
All analytics of the RCT Duplicate project will be run on the Aetion Evidence Platform, which has been tapped by Harvard University and Brigham and Women’s Hospital before as part of Harvard’s REPEAT program aimed at using evidence from datasets to set standards.
According to Aetion, the RWE platform can analyze real-world data (RWD) and RWE to determine medical needs.
The FDA will be given direct access to all study components, process, and results while Aetion provides training on its platform to enable members of the regulatory agency to review completed RWE studies.
Project decides use of RWE for regulatory decisions
The project will identify the ways in which RWE from health care databases can provide robust estimates of treatment safety and efficacy for supplemental new drug applications (NDAs).
Aetion told us that the project’s goal is to provide the FDA with the capability to decide if RWE is “scientifically valid, and where and when it should be used in regulatory decisions.”
The spokesperson explained, “This project has the potential to significantly expand real-world evidence's adoption in health care, which is important because RWE can include patients not currently represented in the clinical trials that drive today's regulatory decisions.”
RCT Duplicate is part of the FDA’s Real-Word Evidence Program which spun out of the 21st Century Cures Act.
While RCT Duplicate is funded by the FDA’s Center for Drug Evaluation and Research (CDER) it was granted additional funding by the National Heart, Lung and Blood Institute at the National Institutes of Health.
Interim results of the study are expected in mid-2019 and full results at the end of 2020.