Overview
As the Centers for Medicare and Medicaid Services (CMS) increasingly use real-world evidence to inform drug price negotiations under the Inflation Reduction Act, understanding how methodological choices impact these analyses is critical. This study evaluated the comparative effectiveness of first-line pembrolizumab versus alternative therapies in advanced non-small cell lung cancer (aNSCLC) among Medicare-eligible patients.
Using a large, deidentified electronic health record-derived dataset from 2011–2023, researchers analyzed outcomes for stage IV aNSCLC patients across three clinical subgroups, applying advanced statistical methods to adjust for differences between treatment groups. The primary outcomes were real-world progression-free survival and overall survival, with scenario analyses exploring how different analytical decisions—such as time period selection, biomarker inclusion, and comparator definitions—affected results.
Why this matters
The study found that survival benefits of pembrolizumab therapies varied depending on methodological choices, with some scenarios showing significant improvements and others showing no difference compared to alternatives. These findings highlight the importance of transparent reporting and rigorous scenario analyses when using real-world data to inform policy decisions like CMS drug price negotiations. By demonstrating how analytical decisions can influence results, this research underscores the need for clear standards to ensure real-world evidence is both reliable and relevant for guiding healthcare policy and improving patient outcomes in the Medicare population.