Characteristics of Real-World Study: Taking the Observational Study of Osimertinib in Patients with EGRF Mutation-Positive NSCLC as an Example

Although the highest level of evidence in evidence-based medicine is randomized controlled trials (RCTs), its universality and external validity are limited due to the strict eligibility criteria and standard treat-ment procedures. Taking the recent observational research of osimertinib in patients with non-small-cell lung cancer (NSCLC) that has an epidermal growth factor receptor (EGFR) mutation as an example, this study deeply analyzes the characteristics of real-world study (RWS), which is an effective supplement to clinical studies, and discusses how to use this real-world evidence (RWE) for evidence-based medical decision-making. The PubMed and Web of Science databases were reviewed for published literature on observational studies of osimertinib for the treatment of NSCLC with EGFR mutations published from inception to April 10, 2022. Independent evaluations of the literature, data extraction, and cross-referencing were carried out by two researchers. A total of 21 studies were included, most of which were uncontrolled, including 15 multi-center studies and 6 single-center studies. We organized content research in the literature, and find that RWS can comprehensively validate the potency of osimertinib in real-world clinical practice, and can provide validation on the effectiveness of osimertinib in the patient population not regularly included or inadequately included in the RCTs, as well as long-term safety data. RWS can potentially identify rare or late-occurring adverse events with Osimertinib. RWS can also optimize real-world sequential treatment strategies with osimertinib. Although RWS cannot attain the superior internal accuracy of RCTs, high-quality RWE can provide valuable additional clinical practice insights for routine care, overcome the deliverability issues of RCTs, and also de-liver complementary information for the generalizability of their results.