FDA to Test Predictive Analytics Against Ongoing Clinical Trials

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Brigham and Women’s Hospital and the U.S. Food and Drug Administration said this month they will expand a program designed to evaluate whether analytical models can successfully replicate the outcomes of clinical trials.

The demonstration project was initially designed to determine whether the use of real-world evidence (RWE) could match the results of 30 completed clinical trials. Now, under the expansion of the program, the technology will also be put to test in real time, as the FDA explores the model’s ability to predict the results of seven ongoing Phase IV 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,” said Jessica Franklin, Ph.D., assistant professor of medicine at Harvard Medical School, biostatistician at Brigham and Women’s Hospital, in a press release.

Franklin said the goal is to get RWE-based technology platforms to the point where they can meet high regulatory standards. The demonstration project is called RCT (Randomized Controlled Trial) Duplicate. It will be run using the Aetion Evidence Platform. Aetion is a New York-based health-tech company founded by faculty from Harvard Medical School.

Part of meeting high regulatory standards is providing regulators with transparency. Aetion said the seven studies will be registered at ClinicalTrials.gov, and regulators will have access to all study components, processes and results. Aetion will also train FDA staff to use and understand the technology.

In an email to Inside Digital Health™, a company spokesperson said one of the reasons for the expansion of the program to include contemporaneous studies is the concern that platform might be manipulated to match the results of completed studies. By putting the platform to the test against studies for which no results are available, the company, investigators and FDA regulators believe they can demonstrate the efficacy of RWE-based prediction while allaying concerns of bias.

The project is part of the FDA’s Real-World Evidence Program, a program mandated by the 2016 21st Century Cures Act. RCT Duplicate is being funded by the FDA’s the Center for Drug Evaluation and Research and led by the agency’s Office of Medical Policy.

In addition to the FDA programs, RCT Duplicate researchers are also working with the National Heart, Lung and Blood Institute at the National Institutes of Health to analyze 25 cardiovascular studies (including both ongoing and completed trials).

If the technology is validated, it could pave the way for drug developers to apply for approval for new products based on simulated, rather than real-world, clinical trials.

“The FDA’s standards will signal to the biopharma industry under what circumstances they could potentially use real-world evidence in regulatory submissions, and what constitutes regulatory-grade RWE,” the Aetion spokesperson said. “Having that certainty is critical to expanding RWE's use.”

In the meantime, although the idea of using RWE is new for regulators, drug developers themselves are already using the technology. Aetion says nine of the top 15 biopharma companies in the world are already using its platform.

“Using the Aetion Evidence Platform, we help them optimize the development and impact of treatments, determine new indications for marketed drugs, advance regulatory approvals, improve access to drugs and engage payers in new ways,” the company said.

Aetion said it expects interim results from the seven clinical trials to be available later this year and final results to come by the end of 2020.

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