FDA expands real-world evidence partnership with Brigham and Women’s Hospital and Aetion
RCT DUPLICATE adds new studies to inform FDA — the first to use real-world evidence to predict treatment safety and efficacy
NEW YORK, April 10, 2019 /PRNewswire/ -- Aetion today announced the U.S. Food & Drug Administration (FDA) and Brigham and Women's Hospital have expanded their demonstration project, RCT DUPLICATE, using real-world evidence to predict the results of seven ongoing Phase IV trials. This is the first time researchers will estimate the results of randomized controlled trials that have not yet concluded.
Building on the program's original scope to use real-world evidence (RWE) to attempt to replicate the results of 30 completed randomized controlled trials (RCTs), the project will inform the FDA's standards for using RWE in regulatory decision-making. Specifically, it will identify when and where RWE based on health care databases can provide robust estimates of treatment safety and efficacy for supplemental new drug applications. The demonstration project is a critical part of the FDA's Real-World Evidence Program, which was mandated by the 21st Century Cures Act.
"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, 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."
RCT DUPLICATE researchers are running all analytics on the Aetion Evidence Platform®.
"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."
To support full transparency, researchers will register the 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.
Now in its implementation phase, RCT DUPLICATE anticipates interim results in mid-2019 and full results at the end of 2020. The researchers have already registered an initial pilot study, which will inform the forthcoming seven studies. The demonstration project is funded by the FDA's Center for Drug Evaluation and Research (CDER) and is led by FDA's Office of Medical Policy (OMP).
In addition, through a grant funded by the National Heart, Lung, and Blood Institute at the National Institutes of Health, RCT DUPLICATE researchers are conducting real-world data analyses on more than 25 cardiovascular studies — ongoing Phase IV trials and published Phase III/IV trials.
Real-world data includes health data routinely collected outside of a controlled setting from insurance claims, electronic medical records, patient-reported outcomes, and patient registries and other sources. Biopharma increasingly uses real-world data for drug safety and efficacy analysis, and FDA has recognized that these data could potentially support regulatory decision-making — the basis for the RCT DUPLICATE project.
Aetion is a health care technology company that delivers real-world evidence for life sciences companies, payers, at-risk providers, and regulatory agencies. The Aetion Evidence Platform analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on treatments, costs, and outcomes. Founded by Harvard Medical School faculty with decades of experience in epidemiology and health outcomes research, Aetion informs health care's most critical decisions — what works best, for whom, and when — to guide treatment development, commercialization, and payment innovation into health care's modern era.
Aetion is based in New York City, and backed by investors including New Enterprise Associates (NEA), Flare Capital Partners, Lakestar, Town Hall Ventures, McKesson Ventures, Sanofi Ventures, Amgen Ventures, UCB, and Horizon Health Services, Inc. Learn more at aetion.com, and follow us at @aetioninc.