Novel framework for the validation of intermediate clinical endpoints in oncology real-world data

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ID: 333126
2026
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Abstract
INTRODUCTION: The role of real-world data in oncology has been substantially increasing, helping to answer clinically unmet needs. No valid framework for real-world data is currently available for the evaluation of surrogate endpoints, with no intermediate clinical endpoint (ICE) being validated as a surrogate for overall survival (OS) across most cancer types. METHODS: A real-world retrospective cohort study was conducted using the nationwide US Flatiron Health database. Four ICEs were evaluated real-world progression-free survival (rwPFS), and three exposure-derived endpoints: Time to Next line of Therapy (TTNT), Time to Last Treatment Administration (TTLA) of first line therapy, and Time to End of First-Line Therapy (TTEFL). Two conditions were evaluated to establish surrogacy: correlation between ICE and OS (condition 1), and correlation of group effects (condition 2). RESULTS: A total of 19,164 patients with metastatic breast cancer (MBC) were included. Median OS was 36.3 (95%CI, 35.6-37.2) months across the study population. For condition 1, a high correlation was found between each of rwPFS, TTNT and TTLA, and OS (Kendall's tau [τ] ≥ 0.5; R2 ≥ 0.7 between 36-month OS and 12-month rates for each ICE across subgroups of interest). For condition 2, group effects were found to be strongly correlated only between rwPFS and OS (R2 ≥ 0.7 across each subgroup comparison). CONCLUSION: Among patients with MBC, rwPFS is a strong surrogate for OS. This study establishes a novel framework applicable to real-world data for the validation of intermediate clinical endpoints. This study can serve as a blueprint for future efforts of real-world data analysis.
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Authors Chris Labaki, Ziad Bakouny, Thibaut Sanglier, Eddy Saad, Miguel Valero Martin, Paolo Tarantino, Andrew Lachlan Schmidt, Jinjoo Shim, David A. Braun, Fanny Bouquet, Maureen Ann Joyce, Marc Machaalani, Eliezer M. Van Allen, Wanling Xie, Sara M. Tolaney, Toni K. Choueiri
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Journal JNCI Journal of the National Cancer Institute
Year 2026
DOI
10.1093/jnci/djag390
URL
https://doi.org/10.1093/jnci/djag390
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