The real-world evidence project at the FDA adds Syapse to its list of partners in a push past trial boundaries
The FDA is making good on its plans to expand the amount and type of real-world evidence it has on hand to help guide its oversight of marketed drugs and push far past the boundaries of clinical data.
The Oncology Center of Excellence is teaming up with San Francisco-based Syapse to expand beyond electronic health records — or EHRs — in building a multi-sourced program involving data drawn from EHRs and registries, as well as molecular data from testing labs.
They also plan to evaluate “real-world endpoints for solid tumors and hematological malignancies, characterizing the usage and clinical impact of molecular testing, understanding outcomes and adverse events in patients receiving precision medicines relative to clinical trial populations, and incorporating patient-reported outcomes into RWE.” And they’ll be working with oncologists on joint outcomes research.
Ex-FDA commissioner Scott Gottlieb made the use of real-world evidence a key part of the agency’s mandate, outlining plans to gather data on marketed drugs to better evaluate proposed label changes as well as hunt for unknown safety factors that should guide prescribing.
“Real-world evidence from well-designed studies meeting appropriate data quality standards can help to inform decision-making and provide information regarding the impact of new therapies in real-world patient populations, particularly those not represented in clinical trials,” said Sean Khozin, associate director for oncology regulatory science and informatics in the FDA’s Oncology Center of Excellence. “This is especially critical in precision medicine, where understanding all of the factors that may drive safety and response is both imperative and difficult to capture at scale using traditional clinical trials.”
Syapse joins COTA and Brigham and Women’s Hospital in working with the agency on real world data. The FDA and Brigham and Women’s Hospital are expanding a pact regulators have with Aetion to see if they can use RWE — one of the buzziest of acronyms in biopharma — to predict the results of randomized clinical trials that have yet to wrap up. And they’re using a batch of 7 upcoming studies to test its accuracy.
“Advancing a deeper understanding of real-world endpoints and analytical methodologies is critical to assuring that all stakeholders can have confidence in the quality of evidence produced and accelerating the use of RWE in regulatory decision-making,” noted company founder and president Jonathan Hirsch in a prepared statement. “The network of health systems that Syapse represents offers a unique opportunity to learn from oncology patient journeys and populations not well represented in traditional clinical trials.”