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FDA Expands Evidence Project to Replace Trials

Data and person graphic

(Gerd Altmann, Pixabay)

10 April 2019. The Food and Drug Administration is expanding a demonstration project with a Boston teaching hospital that tests the value of real world evidence to replace some clinical trials. The FDA project begun in May 2018, known as RCT Duplicate, is a partnership with Brigham and Women’s Hospital, a teaching hospital affiliated with Harvard University.

The original RCT Duplicate project is comparing results of actual medical cases to help make regulatory decisions, without resorting to randomized clinical trials, abbreviated RCT. The 21st Century Cures Act, enacted in December 2016, calls for FDA to investigate the potential value of real world evidence for the agency’s regulatory decision making, which led to establishing the RCT Duplicate project.

In RCT Duplicate up to now, FDA and Brigham and Women’s Hospital are assessing large data sets from 3 health insurance databases to find data comparable to 30 completed clinical trials. The project is using software from Aetion, a health care analytics and technology company in New York, to compile and analyze the data. Jessica Franklin, a biostatistician at Brigham and Women’s Hospital and assistant professor of medicine at Harvard Medical School is leading RCT Duplicate.

Under the expanded project, FDA and Brigham and Women’s Hospital will assess real world evidence to predict the outcomes of 7 post-marketing clinical trials already underway. These studies, also known as phase 4 clinical trials, investigate adverse effects in therapies and medical devices already approved by FDA. In some cases, the initial clinical trials providing evidence for FDA approval do not have follow-up periods to assess safety and continued effectiveness over extended periods of time, thus the need for post-marketing trials.

Franklin notes that this expansion of the RCT Duplicate project will be the first time real world evidence, or RWE, is used to predict the outcomes of ongoing clinical trials. “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,” says Franklin in an Aetion statement. “This additional work will be vital as we develop a process model for the implementation of regulatory-grade RWE studies.”

A pilot study of real world evidence compared to a post-marketing trial, in this case an evaluation of treatments for type 2 diabetes, is registered with, and the 7 studies selected for an expanded RCT Duplicate will also be registered. Interim results from RCT Duplicate are expected in mid-2019, with full results planned for the end of 2020.

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Hat tip: Endpoints News

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