1. Validation analysis of a composite <scp>real‐world</scp> mortality endpoint for patients with cancer in the <scp>United States</scp>
- Author
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Nathan C. Nussbaum, Christina M. Parrinello, Anala Gossai, Qianyi Zhang, and Shirley Monroe
- Subjects
medicine.medical_specialty ,Databases, Factual ,Methods Corner ,overall survival ,Research Brief ,Medical Oncology ,Sensitivity and Specificity ,National Death Index ,03 medical and health sciences ,0302 clinical medicine ,Predictive Value of Tests ,Neoplasms ,Internal medicine ,medicine ,Overall survival ,Electronic Health Records ,Humans ,030212 general & internal medicine ,Sensitivity analyses ,Data Management ,Data source ,business.industry ,030503 health policy & services ,Health Policy ,observational data ,Cancer ,real‐world evidence ,real‐world data ,medicine.disease ,mortality ,Predictive value ,United States ,Cohort ,0305 other medical science ,business ,Social Security Death Index - Abstract
Objective We expanded the previous assessment of a mortality variable suited for real‐world evidence‐focused oncology research. Data source We used a nationwide electronic health record (EHR)‐derived de‐identified database. Data collection We included patients with at least 1 of 18 cancer types between January 1, 2011 and December 31, 2017. Patient‐level structured data (EHRs, obituaries, and Social Security Death Index) and unstructured EHR data (abstracted) were linked to generate a composite mortality variable. Study design We benchmarked sensitivity, specificity, positive predictive value (PPV), negative predictive value (NPV), and ±15‐day agreement against the National Death Index (NDI). Real‐world overall survival (rwOS) was estimated using the Kaplan‐Meier method. We performed sensitivity analyses using a smaller patient cohort that underwent next‐generation sequencing testing. Principal findings Compared with the NDI across 18 cancer types (overall N = 160 436): sensitivity, 83.9%‐91.5% (17/18 cancer types had sensitivity ≥85.0%); specificity, 93.5%‐99.7%; PPV, 96.3%‐98.3%; NPV, 75.0%‐98.7%; ±15‐day agreement, 95.6%‐97.6%; and median rwOS estimates ranging from 2.8% to 12.7% greater. Sensitivity analysis results (n = 17 540) were consistent with the main analysis. Conclusions Across all cancer types analyzed, this composite mortality variable showed high sensitivity, specificity, PPV, NPV, and ±15‐day agreement, and yielded median rwOS values modestly overestimated when compared to NDI‐based results.
- Published
- 2021