ArticlePharmacoEconomics2025
Key Considerations for Assessing Real-World Comparative Effectiveness in the Context of the Drug Price Negotiation Program: A Case Study of Pembrolizumab.
Article in PharmacoEconomics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
BACKGROUND AND
objectiveThe Centers for Medicare and Medicaid Services increasingly rely on real-world evidence to inform drug price negotiations under the Inflation Reduction Act. This study aims to evaluate methodological decisions that impact real-world comparative effectiveness outcomes using a case example of first-line pembrolizumab versus therapeutic alternatives in advanced non-small cell lung cancer among the Medicare population.
methodsThis study used a deidentified, electronic health record-derived, advanced non-small cell lung cancer dataset (2011-23) to analyze Medicare-eligible stage IV patients in three indications: (1) non-squamous, epidermal growth factor receptor, and anaplastic lymphoma kinase negative; (2) squamous; and (3) epidermal growth factor receptor and anaplastic lymphoma kinase negative with programmed death ligand-1 expression ≥1%. Indications (1)-(2) involved pembrolizumab combinations, while (3) referred to pembrolizumab monotherapy. Comparators included common non-platinum-based chemotherapy regimens. Propensity score-based inverse probability weighting was applied. The primary outcomes were real-world progression-free survival and overall survival. Scenario analyses examined the influence of time period selection, programmed death ligand-1 inclusion, therapeutic alternatives, and treatment switching on comparative effectiveness estimates.
resultsIn the non-squamous cohort (1), overall survival benefits of pembrolizumab therapies compared to alternatives varied from a non-significant difference to an improvement of 2.7 months (95% confidence interval 1.2, 4.8), depending on analytical choices. In the squamous cohort (2), pembrolizumab combinations consistently demonstrated overall survival benefits, which ranged from 1.4 months (95% confidence interval 0.1, 3.0) to up to 3.6 months (95% confidence interval 0.1, 5.9). However, for pembrolizumab monotherapy (3), overall survival differences were statistically non-significant. Scenario analyses indicated substantial variability in outcomes based on methodological choices.
conclusionsThis study underscores the importance of transparent reporting and scenario analyses in real-world evidence to support Centers for Medicare & Medicaid Services decision making during drug price negotiations. Findings highlight the need for rigorous methodological standards to ensure the external validity of real-world evidence and its alignment with clinical practice.
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