Augmenting control arms with real-world data for cancer trials: Hybrid control arm methods and considerations.

Background: Hybrid controlled trials with real-world data (RWD), where the control arm is composed of both trial and real-world patients, could facilitate research when the feasibility of randomized controlled trials (RCTs) is challenging and single-arm trials would provide insufficient information.

Methods: We propose a frequentist two-step borrowing method to construct hybrid control arms. We use parameters informed by a completed randomized trial in metastatic triple-negative breast cancer to simulate the operating characteristics of dynamic and static borrowing methods, highlighting key trade-offs and analytic decisions in the design of hybrid studies.

Real-World Outcomes of Patients with Metastatic Non-Small Cell Lung Cancer Treated with Programmed Cell Death Protein 1 Inhibitors in the Year Following U.S. Regulatory Approval.

Background: Evidence from cancer clinical trials has strong internal validity but can be difficult to generalize to real-world patient populations. Here we analyzed real-world outcomes of patients with metastatic non-small cell lung cancer (mNSCLC) treated with programmed cell death protein 1 (PD-1) inhibitors in the first year following U.S.

Development and Validation of a High-Quality Composite Real-World Mortality Endpoint.

Objective: To create a high-quality electronic health record (EHR)-derived mortality dataset for retrospective and prospective real-world evidence generation.

Data Sources/study Setting: Oncology EHR data, supplemented with external commercial and US Social Security Death Index data, benchmarked to the National Death Index (NDI).

Study Design: We developed a recent, linkable, high-quality mortality variable amalgamated from multiple data sources to supplement EHR data, benchmarked against the highest completeness U.

Characteristics of Real-World Metastatic Non-Small Cell Lung Cancer Patients Treated with Nivolumab and Pembrolizumab During the Year Following Approval.

Background: Evidence from cancer clinical trials can be difficult to generalize to real-world patient populations, but can be complemented by real-world evidence to optimize personalization of care. Further, real-world usage patterns of programmed cell death protein 1 (PD-1) inhibitors following approval can inform future studies of subpopulations underrepresented in clinical trials.

Materials And Methods: We performed a multicenter analysis using electronic health record data collected during routine care of patients treated in community cancer care clinics in the Flatiron Health network.

Opportunities and challenges in leveraging electronic health record data in oncology.

The widespread adoption of electronic health records (EHRs) and the growing wealth of digitized information sources about patients is ushering in an era of 'Big Data' that may revolutionize clinical research in oncology. Research will likely be more efficient and potentially more accurate than the current gold standard of manual chart review studies. However, EHRs as they exist today have significant limitations: important data elements are missing or are only captured in free text or PDF documents.