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The Food and Drug Administration (FDA) adopted the ICH E9(R1) guideline on estimands and sensitivity analysis in clinical trials in 2021. The ICH E9(R1) guideline has substantially reshaped the planning and evaluation of confirmatory studies by offering the estimand framework for addressing intercurrent events. Its implementation has led to more transparency about the targeted treatment effects in clinical trials across therapeutic areas. This chapter discusses the FDA’s implementation of the estimand framework across various guideline documents, including treatments for acute myeloid leukemia, chronic rhinosinusitis with nasal polyps, COVID-19, and eosinophilic esophagitis. In these guidelines, the five estimand attributes, along with common intercurrent events in specific disease areas and estimand strategies, are discussed. These insights contribute significantly to trial design, data collection, and selection of analysis methods.

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FDA Guidelines and Their Relationship to the Estimand Framework

  • 章节

  • 发表日期：

    2025年

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  • Wenting Li,
  • Sheinchung Chow,
  • Xue Yang,
  • Qiong Du,
  • Alice Wang,
  • Na Yang,
  • Liwen Liang

摘要

The Food and Drug Administration (FDA) adopted the ICH E9(R1) guideline on estimands and sensitivity analysis in clinical trials in 2021. The ICH E9(R1) guideline has substantially reshaped the planning and evaluation of confirmatory studies by offering the estimand framework for addressing intercurrent events. Its implementation has led to more transparency about the targeted treatment effects in clinical trials across therapeutic areas. This chapter discusses the FDA’s implementation of the estimand framework across various guideline documents, including treatments for acute myeloid leukemia, chronic rhinosinusitis with nasal polyps, COVID-19, and eosinophilic esophagitis. In these guidelines, the five estimand attributes, along with common intercurrent events in specific disease areas and estimand strategies, are discussed. These insights contribute significantly to trial design, data collection, and selection of analysis methods.