Syapse and the U.S. Food and Drug Administration (FDA) Oncology Center of Excellence (OCE) have signed a multi-year Research Collaboration Agreement (RCA) focused on the use of real-world evidence (RWE) 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.
Methods to Derive RWE From Multiple Sources including EHRs
As part of this research collaboration, Syapse and the FDA will investigate methods to derive RWE from multiple sources including, and going beyond, electronic health records (EHRs). The “Framework for FDA’s Real-World Evidence Program” describes the challenges in obtaining comprehensive data from EHRs and claims, and the difficulty in linking sources. This collaboration will utilize real-world data integrated from many source systems, including clinical data from EHRs and registries, and molecular data from testing labs, and seek to characterize the regulatory suitability of RWE derived from this multi-source approach.
RWE Use to Support Precision Medicine & Oncology
Additionally, Syapse and the FDA will examine 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.
Alongside these direct collaboration efforts, Syapse will engage oncologists in its Learning Health Network in joint outcomes research. This work will support Syapse in enabling the use of RWE 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.
Why It Matters
“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’s Oncology Center of Excellence. “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.”
