Abstract 07: Use of a real-world data registry to rapidly generate outcomes data following a case study of a novel treatment combination in pancreatic adenocarcinoma

XCELSIOR is an IRB-approved, patient-centric, real-world data and outcomes registry for developing operational and analytic methods in precision oncology. Earlier this year, Kinsey et al. reported on an exceptional response in a heavily pretreated pancreatic adenocarcinoma patient using combined MEK inhibition with autophagy inhibition (Nature Medicine 2019). We sought to understand the utilization of this regimen in clinical practice and analyze safety and outcomes information from patients in XCELSIOR identified as being treated with this combination. Searching the XCELSIOR database, we identified eleven patients for whom this regimen had been considered, four patients at different centers who received this regimen as part of their clinical care for pancreatic adenocarcinoma, and two more patients expected to start treatment soon. As part of their participation in XCELSIOR, these patients shared access to their full medical records, which were collected, processed, and abstracted into a 21 CFR 11 compliant database for analysis. We intend to present demographics, safety, biomarkers, and survival data on these patients and five additional patients treated with this combination at the Huntsman Cancer Center. For late-stage cancer patients, a patient-centric outcomes registry can provide continuous high-quality, real-world data to rapidly identify safety and outcomes information for cancer patients treated in clinical practice with novel and potentially efficacious off-label combinations, and to aggregate that data into a high-quality dataset for further Bayesian analysis. Citation Format: Lola Rahib, Mark Shapiro, Jeff Shrager, Bryan Federowics, Conan Kinsey. Use of a real-world data registry to rapidly generate outcomes data following a case study of a novel treatment combination in pancreatic adenocarcinoma [abstract]. In: Proceedings of the AACR Special Conference on Advancing Precision Medicine Drug Development: Incorporation of Real-World Data and Other Novel Strategies; Jan 9-12, 2020; San Diego, CA. Philadelphia (PA): AACR; Clin Cancer Res 2020;26(12_Suppl_1):Abstract nr 07.