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Response- and progression-based endpoints in trial and observational cohorts of patients With NSCLC


May 2024


Lu Y, Langerman SS, McCain E, et al. Response- and progression-based endpoints in trial and observational cohorts of patients With NSCLC. JAMA Network Open. 2024.


Real-world evidence (RWE) is increasingly valuable in understanding treatment patterns and outcomes of people receiving cancer care outside the clinical trial setting, utilizing data from various sources like electronic health records (EHRs) and claims. These insights complement traditional clinical trial data and can be used to inform drug development and clinical decision making.

In oncology, regulators often use overall survival as an endpoint for approval, but the rise of accelerated approval means proxy or surrogate endpoints like progression-free survival (PFS) and response rates are increasingly used. In solid tumor studies, typically the RECIST v1.1 guideline is used to determine response and progression. However, assessing treatment effectiveness in routine care relies on clinician judgment rather than standardized criteria like RECIST v1.1.

Previous research has shown the feasibility of capturing real-world measures of effectiveness from clinician documentation in the EHR, like real-world progression (rwP) and real-world response (rwR), and their ability to provide valuable insights. While previous investigations have compared real world and clinical trial outcomes among aligned patient cohorts, there has been limited opportunity to leverage patient level clinical trial and RWD to conduct these studies. 

In this study, researchers from Genentech and Flatiron Health collaborated to compare response-based endpoints including response rate, duration of response and progression-free survival, from a clinical trial dataset utilizing RECIST v1.1 with a real-world data set utilizing clinician-documented rwR and rwP, to generate evidence as to the relationship between these outcome measures and further enhance confidence in real-world outcome variables.

Why this matters

This study holds significant importance as it demonstrates consistency between real-world and clinical trial endpoints, such as objective response rate (ORR/rwRR), duration of response (DOR/rwDOR), and progression-free survival (PFS/rwPFS), in a cohort of patients with stage IV non-small cell lung cancer. Despite inherent differences between clinical trials and routine care, the findings shed light on crucial methodological considerations for interpreting clinical outcomes across these settings. Moreover, the study's analytical framework provides a blueprint for evaluating endpoint comparability in other disease contexts, potentially bridging the gap between real-world and trial data.

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