Healthcare data firm Syapse has begun working with the FDA to help bring real-world evidence to bear on the agency’s decisions to approve cancer drugs.
The multiyear research collaboration with the FDA’s Oncology Center of Excellence will have a special focus on precision medicine and the evaluation of targeted therapies. In addition, Syapse and agency staff aim to build a framework for answering key regulatory questions regarding potential cancer therapies’ safety, dosing and patient outcomes, as well as ways to identify patterns in testing and treatment.
“Advances in real-world evidence present an opportunity to learn from patients and potentially translate those insights into safer and more effective therapies,” the FDA’s Sean Khozin, associate director for oncology regulatory science and informatics, said in a statement.
“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,” Khozin added. “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 and the FDA will also explore the use of real-world endpoints for cancer treatments, such as the clinical impact of molecular testing, as well as the incorporation of patient-reported outcomes into real-world evidence. The two plan to tap multiple data sources, including from electronic health records, registries and more, plus molecular diagnostic data.
“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,” said Syapse founder and President Jonathan Hirsch.
Syapse recently inked a deal with Pfizer to help provide precision medicine data gathering and real-world outcomes research for its work in cancer, focusing on the factors that may influence oncologists as they choose different molecular testing and therapy strategies for different patients.