FDA is partnering with Aetion (NY, USA) and Brigham and Women’s Hospital (MA, USA) in this ground-breaking project.
Building on a previous project to replicate 30 randomized controlled trials, RCT DUPLICATE, which used the Aetion Evidence Platformâ„¢ (NY, USA), is being expanded to use real-world evidence (RWE) to predict the results of seven Phase IV trials. This is the first time researchers will attempt to predict the results of trials that have not concluded.
“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, assistant professor of medicine at Harvard Medical School, biostatistician at Brigham and Women’s Hospital (both MA, USA), 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.”
The results of these new trials are expected in mid-2019, with full results coming at the end of 2020. The project is being funded by FDA’s Center for Drug Evaluation and Research (CDER) and is led by FDA’s Office of Medical Policy (OMP). It is hoped these results will inform the development of FDA standards into the use of RWE in regulatory decision-making.
“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 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.”