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Regulatory Focus – FDA’s OCE Taps Syapse for Research on RWE

Regulatory Focus – FDA’s OCE Taps Syapse for Research on RWE

San Francisco-based data company Syapse said Wednesday that it has signed a multi-year research collaboration agreement with the US Food and Drug Administration’s (FDA) Oncology Center of Excellence (OCE) to work on using real-world evidence (RWE) to support regulatory decision making.


Syapse and OCE will work with EHRs, labs and registries, among other data sources, to characterize the regulatory suitability of RWE derived from multiple sources. They will also work together to examine real-world endpoints for solid tumors and hematological malignancies, characterizing the usage and clinical impact of molecular testing, as well as understanding outcomes and adverse events relative to clinical trial populations, and incorporating patient-reported outcomes into RWE.

Sean Khozin, associate director for oncology regulatory science and informatics in OCE, 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.”


The collaboration will build on FDA’s work in using RWD and RWE to expand indications for already approved treatments and use RWE to inform effectiveness data. In April, FDA approved a new indication for Pfizer’s Ibrance (palbociclib) based on data from electronic health records and postmarketing reports of Ibrance in male patients sourced from three databases.


Also in April, FDA and Brigham and Women’s Hospital expanded a demonstration project using RWE to predict the results of seven ongoing Phase IV trials.


And OCE is working with Flatiron Health, Friends of Cancer Research, the National Cancer Institute and other stakeholders on the use of RWE.


“OCE is also working on a project exploring whether it’s possible to use real world endpoints, like time to treatment discontinuation (TTD) from legacy datasets as a potential real-world endpoint for pragmatic randomized clinical trials for FDA-approved therapies in the off-label setting, and whether synthetic control arms based on prior clinical trials can be used for tumor types where the standard of care has not changed in decades, and the prognosis is especially poor,” former FDA Commissioner Scott Gottlieb said in January.…