FDA Expands Real-World Evidence Partnership with Brigham and Women’s

The FDA, Brigham and Women’s Hospital, and Aetion are expanding their real-world evidence (RWE) demonstration to predict the results of seven ongoing FDA drug trials.

The FDA drug or treatment trials are in Phase IV, meaning the drug or treatment is being tracked over a long period for a greater number of patients. The partnership between the three organizations, called RCT DUPLICATE, marks the first time researchers will estimate the results of randomized controlled trials that haven’t yet finished.

The expanded partnership will build on the original idea of RCT DUPLICATE, which uses real-world evidence to try to replicate the results of 30 randomized, controlled FDA trials. RCT DUPLICATE will inform the FDA’s standards for using real-world evidence in regulatory decisions. It will also show the FDA when and where RWE can provide high-quality estimates of the safety and effectiveness for supplemental new drug applications.

“The project will play a major role in determining the future of how drugs are developed and approved,” said Aetion CEO Carolyn Magill. “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. By extending this project, FDA shows its commitment to understanding how best to incorporate real-world evidence into regulatory decisions.”

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

Real-world evidence refers to health data that is routinely collected outside of a controlled setting. This can include insurance claims, electronic health records, patient-reported outcomes, patient registries, and other sources. The biopharma industry is using RWE for drug safety and effectiveness analysis, and the FDA has recognized that this data could be used to support regulatory decision-making.

“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 at Harvard Medical School, biostatistician at Brigham and Women’s Hospital, and lead of RCT DUPLICATE. “This additional work will be vital as we develop a process model for the implementation of regulatory-grade RWE studies.”

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