Abstract
While randomized controlled trials (RCTs) are the gold standard for evidence-based medicine, they do not always reflect real-world patient populations, limiting their generalizability and external validity. Real-world evidence (RWE), generated during routine clinical practice, is increasingly important in determining effectiveness outside of the tightly controlled conditions of RCTs, and is now recognized by regulatory bodies as a valuable complement to RCTs. Consequently, it is increasingly important for physicians to understand how RWE data can be used alongside clinical trial data. Here, we discuss the different types of real-world observational studies, outline the benefits and limitations of RWE, and, using examples from EGFR mutation-positive non-small-cell lung cancer, outline how RWE can be used to help inform treatment decisions.
Keywords:
EGFR TKIs; EGFR mutation-positive; NSCLC; real-world.
MeSH terms
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Carcinoma, Non-Small-Cell Lung / genetics
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Carcinoma, Non-Small-Cell Lung / mortality
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Carcinoma, Non-Small-Cell Lung / therapy*
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Chemotherapy, Adjuvant / methods
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Chemotherapy, Adjuvant / statistics & numerical data
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Data Interpretation, Statistical
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Electronic Health Records / statistics & numerical data
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ErbB Receptors / antagonists & inhibitors
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ErbB Receptors / genetics
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Evidence-Based Medicine / statistics & numerical data*
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Gain of Function Mutation
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Humans
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Lung Neoplasms / genetics
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Lung Neoplasms / mortality
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Lung Neoplasms / therapy*
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Medical Oncology / methods
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Medical Oncology / statistics & numerical data
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Medical Oncology / trends
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Pneumonectomy
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Precision Medicine / methods
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Precision Medicine / statistics & numerical data
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Precision Medicine / trends
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Progression-Free Survival
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Protein Kinase Inhibitors / pharmacology
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Protein Kinase Inhibitors / therapeutic use*
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Randomized Controlled Trials as Topic / statistics & numerical data
Substances
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Protein Kinase Inhibitors
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EGFR protein, human
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ErbB Receptors