FDA Expands RWE Partnership with Brigham and Women’s Hospital and Aetion

Aetion, Inc announced the U.S. Food & Drug Adminsitration (FDA) and Brigham Women's Hospital have expanded their demonstration project, RCT DUPLICATE, using real-world evidence to predict the results of seven ongoing Phase IV trials. This is the first time researchers will estimate the results of randomized controlled trials that have not yet concluded.

Building on the program's original scope to use real-world evidence (RWE) to attempt to replicate the results of 30 completed randomized trials (RCTs), the project will inform the FDA's standards for using RWE in regulatory decision-making. Specifically, it will identify when and where RWE based on healthcare databases can provide robust estimates of treatment safety and efficacy for supplemental new drug applications. The demonstration project is a critical part of the FDA's Real-World Evidence Program, which was 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, Ph.D., assistant professor of medicine at Harvard Medical School, biostatistician at Brighan and Women's Hospial, and lead of RCT DUPLICATE. "This addtional work willbe vital as we develop a process model for the implementaton of regulatory-grade RWE studies."

RCT DUPLICATE researchers are running all analytics on the Aetion Evidence PlatformTM.

"This project will play a major role in determining the future of how drugs are developed and approved," said Carolyn Magill, CEO of Aetion. "Data collected from the front lines of clinical care can often provide a more complete picture of how treatments affect patients not represented in clinical trials, but the regulatory process must ensure transparency. By extending this project, FDA shows its commitment to understanding how to best to incorporate real-world evidence into regulatory decisions."

To support full transparency, researchers will register the seven additional studies on clinicaltrials.gov, and give FDA direct access to all study components, processes, and results. Aetion is training FDA staff on is platform to enable them to work with its techonolgy to review a completed RWE study and test its robustness.

Now in its implementation phase, 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. The demonstration project is funded by the FDA's Center for Drug Evaluation and Research (CDER) and is led by FDA's Office of Medical Policy (OMP).

In addition, through a grant fundedb the National Heart, Lung, and Blood Institute at th National Insitutes of Heath, RCT DUPLICATE researchers are conducting real-world data analyses on more than 25 cardiovascular studies-ongoing Phase IV trials and published Phase III/IV trials.

Real-world data includes health data routinely collected outside of a controlled setting from insurance claims, electronic medical records, patient-reported outcomes, and patient registries and other sources. Biopharma increasingly uses real-world data for drug safety and efficacy analysis, and FDA has recognized that these data could potentially support regulatory decision making-the basis for the RCT DUPLICATE project.