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Abstract:
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Overall survival (OS) is an important outcome measure in oncology, but it may require large sample sizes and long follow-up time to provide meaningful insights. As a supplement to OS, outcomes based on disease progression and tumor response typically require less follow-up time and are commonly captured in oncology clinical trials to assess treatment efficacy. The standard methods to collect these outcomes in clinical trials, however, may not be feasible using electronic health records (EHR), but such data present an opportunity to answer research questions at a scale and recency not available from clinical trials, reflecting treatment patterns and populations seen in routine clinical practice. As we develop real-world versions of these outcomes, it is critical to apply a statistical framework to assess their quality. We illustrate such a framework with a case study evaluating the reliability and validity of real-world progression-free survival in advanced non-small cell lung cancer. We discuss statistical considerations unique to working with EHR data, and identify methodological gaps that present opportunities for future development.
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