Abstract 1676: A hybrid ontology and graph system enabling precision oncology applications through real-time incorporation of omics biomarkers into a scalable real-world data platform

Using activities to create and collect data is not a new idea. Teachers have been incorporating real-world data into their classes since at least the advent of the graphing calculator. Plenty of data collection activities and data sets exist, and the graphing calculator has made modeling data much easier. However, the authors were in search of a better physical model for a quadratic. We wanted students to see an actual parabola take shape in real time and then explore its characteristics, but we could not find such a hands-on model.

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A Real-time Dynamic Simulation Scheme for Large-Scale Flood Hazard Using 3D Real World Data

2007 11th International Conference Information Visualization (IV '07) ◽

10.1109/iv.2007.15 ◽

2007 ◽

Cited By ~ 6

Author(s):

C Wang ◽

T. R. Wan ◽

I. J. Palmer

Keyword(s):

Real Time ◽

Dynamic Simulation ◽

Real World ◽

Large Scale ◽

Flood Hazard ◽

Real World Data ◽

World Data ◽

Time Dynamic ◽

Simulation Scheme

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Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development

Frontiers in Oncology ◽

10.3389/fonc.2021.695936 ◽

2022 ◽

Vol 11 ◽

Author(s):

Timothy A. Yap ◽

Ira Jacobs ◽

Elodie Baumfeld Andre ◽

Lauren J. Lee ◽

Darrin Beaupre ◽

...

Keyword(s):

Clinical Trials ◽

Drug Development ◽

Real World ◽

External Control ◽

Control Group ◽

Precision Oncology ◽

Efficacy And Safety ◽

Control Groups ◽

Real World Data ◽

World Data

Randomized controlled trials (RCTs) that assess overall survival are considered the “gold standard” when evaluating the efficacy and safety of a new oncology intervention. However, single-arm trials that use surrogate endpoints (e.g., objective response rate or duration of response) to evaluate clinical benefit have become the basis for accelerated or breakthrough regulatory approval of precision oncology drugs for cases where the target and research populations are relatively small. Interpretation of efficacy in single-arm trials can be challenging because such studies lack a standard-of-care comparator arm. Although an external control group can be based on data from other clinical trials, using an external control group based on data collected outside of a trial may not only offer an alternative to both RCTs and uncontrolled single-arm trials, but it may also help improve decision-making by study sponsors or regulatory authorities. Hence, leveraging real-world data (RWD) to construct external control arms in clinical trials that investigate the efficacy and safety of drug interventions in oncology has become a topic of interest. Herein, we review the benefits and challenges associated with the use of RWD to construct external control groups, and the relevance of RWD to early oncology drug development.

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PMU121 APPLICATION OF REAL-WORLD DATA FROM MEDICAL BIG-DATA PLATFORM IN REAL WORLD EVIDENCE GENERATION: A PRACTICE IN POST-MARKETING RESEARCH IN CHINA

Value in Health ◽

10.1016/j.jval.2020.04.885 ◽

2020 ◽

Vol 23 ◽

pp. S256

Author(s):

Y. Wang ◽

Y. Xing ◽

Y. Wu ◽

N. Yuan ◽

F. Wang ◽

...

Keyword(s):

Big Data ◽

Real World ◽

Marketing Research ◽

Real World Data ◽

World Data ◽

Real World Evidence ◽

Data Platform ◽

Post Marketing ◽

Medical Big Data ◽

Evidence Generation

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Use of a real-world data approach to rapidly generate outcomes data following a case study of a novel treatment combination in pancreatic adenocarcinoma.

Journal of Clinical Oncology ◽

10.1200/jco.2020.38.15_suppl.e16735 ◽

2020 ◽

Vol 38 (15_suppl) ◽

pp. e16735-e16735

Author(s):

Lola Rahib ◽

Karen Chen ◽

Allyson J. Ocean ◽

Changqing Xie ◽

Austin Duffy ◽

...

Keyword(s):

Pancreatic Cancer ◽

Partial Response ◽

Median Time ◽

Real World ◽

Clinical Benefit ◽

Progressive Disease ◽

Metastatic Pancreatic Cancer ◽

Precision Oncology ◽

Real World Data ◽

World Data

e16735 Background: We use a real-world data approach to report on safety and benefits on metastatic pancreatic cancer pts who were treated with a MEK inhibitor plus hydroxychloroquine (HCQ) after exhausting all other treatment options. MEK inhibition acts on the KRAS pathway, which in turn increases autophagy as a resistance mechanism, furthermore, HCQ inhibits autophagy causing a cytotoxic effect. This combination was shown to diminish tumor volume in xenograft mouse models and a partial response in one heavily pre-treated patients was reported. Methods: XCELSIOR is an IRB approved, patient-centric, real-world data and outcomes registry for developing operational and analytic methods in precision oncology. Searching the XCELSIOR database, we identified 14 pts for whom this regimen had been considered. 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 additionally collected de-identified data on 12 pts treated with this combination from five academic centers. Three more patients are expected to start treatment soon. Results: Between March 2018 and January 2020, 15 patients treated with the trametinib/HCQ combination and 3 patients treated with cobimetinib/HCQ were identified in XCELSIOR and five academic institutions. The median age at diagnosis was 64 (range 43-74) and 56% were male. For patients treated with trametinib/HCQ, the median time on treatment was 67 days (range 5-172 days), 11 patients were treated for more than 30 days (median time 97 days). The median PFS for this group was 2.9 months and the median OS was 7.4 months. The clinical benefit rate was 60% for the 10 evaluable patients treated with trametinib/HCQ, 1 patient had a partial response (previously published), 5 had stable disease (for at least 8 weeks) and 4 had progressive disease (physician reported). 2/3 patients treated with cobimetinib/HCQ were on treatment for more than 30 days and all three had progressive disease within 7 weeks. The most common side effects were Grade 1 fatigue and Grade 1/2 rash for both combinations. An additional 3 patients will start treatment soon and will be included in the analysis. Conclusions: Combinatorial MEK and autophagy inhibition was well tolerated in heavily treated metastatic pancreatic cancer patients. Trametinib/HCQ demonstrates some clinical benefit for this group. We demonstrate the feasibility of utilizing real-world data in precision oncology. Clinical trial information: NCT03793088 .

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PO2RDF: Representation of Real-world Data for Precision Oncology Using Resource Description Framework

10.21203/rs.3.rs-219654/v1 ◽

2021 ◽

Author(s):

Yiqing ZHAO ◽

Anastasios Dimou ◽

Feichen Shen ◽

Nansu Zong ◽

Jaime I. Davila ◽

...

Keyword(s):

Real World ◽

Targeted Therapies ◽

Resource Description Framework ◽

Clinical Decision Making ◽

Egfr Mutations ◽

Precision Oncology ◽

Real World Data ◽

World Data ◽

Description Framework ◽

Resource Description

Abstract Background: Next-generation sequencing provides comprehensive information about individuals’ genetic makeup and is commonplace in precision oncology practice. Due to the heterogeneity of individual patient’s disease conditions and treatment journeys, not all targeted therapies were initiated despite actionable mutations. To better understand and support the clinical decision-making process in precision oncology, there is a need to examine real-world associations of patients’ genetic information and treatment choice.Methods: To fill the gap of insufficient use of real-world data (RWD) in electronic health records (EHRs), we generated a single Resource Description Framework (RDF) resource, called PO2RDF (precision oncology to RDF) by integrating information regarding gene, variant, disease, and drug from genetic reports and EHRs. Results: There are total 2,309,014 triples contained in the PO2RDF. Among them 32,815 triples are related to Gene, 34,695 triples are related to Variant, 8,787 triples are related to Disease, 26,154 triples are related to Drug. We performed one use case analysis to demonstrate the usability of the PO2RDF: we examined real-world associations between EGFR mutations and targeted therapies to confirm existing knowledge and detect off-label use. Conclusions: In conclusion, our work proposed to use RDF to organize and distribute clinical RWD that is otherwise inaccessible externally. Our work serves as a pilot study that will lead to new clinical applications and could ultimately stimulate progress in the field of precision oncology.

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The master observational trial–a novel method to unify precision oncology data collection.

Journal of Clinical Oncology ◽

10.1200/jco.2020.38.15_suppl.e19313 ◽

2020 ◽

Vol 38 (15_suppl) ◽

pp. e19313-e19313

Author(s):

Dane J. Dickson ◽

Jennifer Maria Johnson ◽

Raymond C. Bergan ◽

Rebecca Owens ◽

Vivek Subbiah ◽

...

Keyword(s):

Real World ◽

Unmet Needs ◽

Data Availability ◽

Precision Oncology ◽

Real World Data ◽

Observational Trial ◽

World Data ◽

Patient Consent ◽

National Implementation ◽

Real World Datasets

e19313 Background: The Master Observational Trial (MOT) was recently created as a new master protocol that hybridizes the power of master interventional trials with the richness of real-world data (Cell, 2020). The MOT can be described as a series of prospective observational studies that are tied together through a common protocol, infrastructure, and organization. The MOT has broad application in many disease states but is particularly powerful in oncology. We herein expand our prior work to describe key details regarding how the MOT concept can fill multiple unmet needs in oncology. Methods: Through published information, white papers, and expert opinions we identified key unmet needs of oncology stakeholders. We reviewed the publicly available information of structure, organization, and data availability of the five largest genomic-outcome real-world data efforts. Common concerns included variability and reliability of biomarkers, the scientific rigor in real-world data, data silos, patient consent, and duplicated or disparate activities. We then determined how a specific application of the MOT in oncology could answer stakeholder concerns, integrate with current efforts, and also how to provide a model that would be equally valuable to academic and community clinics. Results: We identified significant scientific challenges with many of the current oncology real-world datasets in answering key concerns of stakeholders. We developed the Master Registry of Oncology Outcomes Associated with Testing and Treatment (ROOT) as the first national implementation of an oncology-centric MOT. We modeled how ROOT could fill scientific gaps in current data efforts and integrate with interventional and real-world efforts and help answer key concerns of stakeholders. We also identified solutions that would allow community and academic groups to participate in the same effort. Conclusions: An oncology-centric MOT has the potential to improve the quality of RWD in oncology and advance precision oncology in ways that are not fully addressed by current retrospective efforts. Reference Dickson DJ, Johnson J, Owens R, Bergan R, Subbiah V, Kurzrock R. (2020). The Master Observational Trial: A New Class of Master Protocol to Advance Precision Medicine. Cell 180, 9-14. Clinical trial information: NCT04028479 .

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