Emulation of the CheckMate 017/057 (NCT01642004 and NCT01673867) Trials Using Specialty Oncology Electronic Health Records Databases

Randomized controlled trials (RCTs) are generally regarded as the gold-standard of evidence for establishing efficacy of medical products. However, real-world data (RWD) are increasingly used to complement evidence from RCTs. Yet, to have confidence in the accuracy of non-interventional studies of medical products and their outcomes in oncology, investigators need to know what questions can be validly answered, with which non-interventional study designs, and which analysis methods are appropriate, given the data that is available. Building on a process from the RCT DUPLICATE initiative, EmulatioN of Comparative Oncology trials with Real-world Evidence (ENCORE) is the trial emulation discussed in this protocol is part of the expansion project specific to oncology and aims to emulate 12 randomized oncology RCTs using multiple EHR data sources. The purpose of this protocol is to describe the emulation of the pooled analysis of CheckMate 017 and CheckMate 057. CheckMate 017/057 were two Phase III, double-blind, randomised studies assessing the efficacy and safety of Nivolumab (3 mg/kg IV every 2 weeks) versus Docetaxel (75 mg/m² IV every 3 weeks) in patients with squamous (CheckMate 017) and nonsquamous (CheckMate 057) advanced Non-small Cell Lung Cancer (NSCLC) and disease progression during or after prior platinum-based chemotherapy.