Using real-world data, the FDA aims to predict randomized trial results before they’re done

The project aims to help the FDA systematize the use healthcare databases in estimating a product's safety and efficacy for supplemental new drug applications. (Andrew Harnik, Associated Press)

The FDA is taking new steps to test the capabilities of real-world evidence, by attempting to predict the results of randomized, controlled trials before they are completed.

It’s an awaited expansion of the agency’s demonstration project, currently being conducted with the data firm Aetion and Brigham and Women’s Hospital.

The RCT DUPLICATE program originally looked to replicate 30 previously completed clinical trials using real-world data. Now, it aims to prognosticate the outcomes of seven ongoing postmarket studies—a first for the field, according to Aetion.


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"This project will play a major role in determining the future of how drugs are developed and approved," Aetion CEO Carolyn Magill said in a statement. "Data collected from the front lines of clinical care can often provide a more complete picture on how treatments affect patients not represented in clinical trials, but the regulatory process must ensure transparency."

The project’s ultimate goal, of course, is to assist the FDA’s continuing development of standards for using real-world evidence in regulatory decisions—namely, it will help identify when and where healthcare databases can provide proper estimates of safety and efficacy for supplemental new drug applications.

"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 project lead Jessica Franklin, Ph.D., assistant professor of medicine at Harvard Medical School and a biostatistician at Brigham and Women's Hospital. "This additional work will be vital as we develop a process model for the implementation of regulatory-grade RWE studies." ­

RELATED: Aetion closes on $27M extension of series B financing to expand its real-world evidence platform

Researchers will register the seven additional phase 4 studies on, and give the FDA direct access to all study components, processes and results, according to Aetion, which is training FDA staff on its platform.

In addition—through a grant funded by the NIH’s National Heart, Lung, and Blood Institute—the program is conducting real-world data analyses on more than 25 cardiovascular studies, including ongoing phase 4 trials and published phase 3 and 4 trials.

The RCT DUPLICATE project—mandated by the 21st Century Cures Act and funded by the FDA's Center for Drug Evaluation and Research—expects to see interim results by mid-2019 and full results by the end of 2020.

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