Syapse and the U.S. Food and Drug Administration (FDA) Oncology Center of Excellence (OCE) have signed a multiyear research collaboration agreement focused on the use of real-world evidence to support regulatory decision-making. Syapse and the OCE will work with stakeholders across the FDA to address key regulatory questions about testing and treatment patterns, dosing and safety, and outcomes in oncology with a focus on precision medicine.
Sean Khozin, MD, MPH
“Advances in real-world evidence present an opportunity to learn from patients and potentially translate those insights into safer and more effective therapies,” said Sean Khozin, MD, MPH, Associate Director for Oncology Regulatory Science and Informatics in the FDA OCE. “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. 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.”
Beyond Electronic Health Records
As part of this collaboration, Syapse and the FDA will investigate methods to derive real-world evidence from multiple sources including, and going beyond, electronic health records. The Framework for FDA’s Real-World Evidence Program describes the challenges in obtaining comprehensive data from electronic health records and claims as well as the difficulty in linking sources. This collaboration will utilize real-world data integrated from many source systems—including clinical data from electronic health records and registries and molecular data from testing laboratories—and will seek to characterize the regulatory suitability of real-world evidence derived from this multisource approach.
Additionally, Syapse and the FDA will examine real-world endpoints for solid tumors and hematologic 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 real-world evidence.
Learning Health Network
Along with these direct collaboration efforts, Syapse will engage oncologists in its Learning Health Network, a global precision oncology data-sharing network, in joint outcomes research. This work will support Syapse in enabling the use of real-world evidence by oncology care providers to inform care decisions and outcomes research. In turn, Syapse will incorporate the outcomes derivation methodology into its Learning Health Network capabilities.
“Syapse is proud to collaborate with the FDA to enhance understanding of how patients respond to therapies outside of clinical trials to improve care and outcomes,” said Jonathan Hirsch, Founder and President of Syapse. “Advancing a deeper understanding of real-world endpoints and analytic methodologies is critical to assuring that all stakeholders can have confidence in the quality of evidence produced and accelerating the use of real-world evidence in regulatory decision-making.” ■