Benefits and Limitations of Real-World Evidence: Lessons from EGFR Mutation-Positive Non-Small-Cell Lung Cancer

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.

Evaluation of the effect of triclosan coated sutures in the prevention of surgical site infections in a Spanish hospital setting: A prospective, observational study

“…Foremost, as a real-world study, it permitted the analysis of a broader and more diverse distribution of patients, reflective of clinical practice, than would be practical in an RCT setting. [ 51 , 52 ] Further, as it was unicentric, potential variables such as surgeon experience, SSI prevention measures, and differences across operating rooms were the same in all time periods. In addition, patients were recruited from different surgical specialties, and with different characteristics, facilitating the study of a more representative population.…”

Real-Word Experience of Cabozantinib in Metastatic Renal Cell Carcinoma (mRCC): Results from the Canadian Kidney Cancer information system (CKCis)

“…However, results need to be interpreted in light of a number of limitations. These include the retrospective nature of the cohort, heterogeneity of patient population, missing data, and selection bias [32] In the multivariable analysis, most of the subgroups were underpowered to assess prognostic factors. Furthermore, data on safety outcomes is not available for this manuscript.…”

Prognostic model of long-term advanced stage (IIIB-IV) EGFR mutated non-small cell lung cancer (NSCLC) survivors using real-life data

“…6 Hospital General Universitario de Alicante, 03010 Alicante, Spain. 7 Hospital Clínico San Carlos, 28040 Madrid, Spain. 8 Complexo Hospitalario Universitario A Coruña, 15006 A Coruña, Spain.…”

“…Randomized controlled trials (RCT) are considered the gold standard of evidence-based medicine. However, populations included may not be representative patients in real life considering that in many cases, these are older, have a poorer performance status (PS), rare mutations and with brain metastases detected more frequently at diagnosis [7]. For this reason, patient registry serves as real world data studies to verify the results obtained in RCT rather than carrying out phase IV studies in the clinical setting.…”