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The China National Medical Products Administration (NMPA) adopted the ICH E9(R1) guideline on estimands and sensitivity analysis in clinical trials in early 2021. Considering the growing importance of well-defined estimands in clinical trials, this chapter discusses the NMPA’s implementation of the estimand framework across various guideline documents, focusing primarily on drug development for Crohn’s Disease (CD) and Uclcerative Colitis (UC), real-world studies (RWS), and pediatric extrapolation. We first discuss key principles embedded within the NMPA guidelines for drug development in CD and UC, emphasizing the treatment goal, rigorous trial designs, and considerations for estimands regarding population, variable, treatment, and intercurrent events. The chapter also illustrates the complexities of establishing estimands in RWS, highlighting considerations of diverse patient populations, treatment regimens, and intercurrent events related to behavior and nonbehavioral factors. Drawing attention to recent suggestions for pediatric extrapolation, we emphasize the necessity of clear definitions of estimands aligning with trial objectives. In closure, the chapter ascertains the importance of proactive communication between sponsors and the NMPA to ensure adherence to these guidelines and successful implementation of estimands in China.

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NMPA Guidelines and Their Relationship to the Estimand Framework

  • 章节

  • 发表日期：

    2025年

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  • Yuhan Huang,
  • Jie Chen,
  • Jun Zhao,
  • Xiaoni Liu,
  • Mingli Heng,
  • Yan Hou,
  • Jun Wang,
  • Pingyan Chen

摘要

The China National Medical Products Administration (NMPA) adopted the ICH E9(R1) guideline on estimands and sensitivity analysis in clinical trials in early 2021. Considering the growing importance of well-defined estimands in clinical trials, this chapter discusses the NMPA’s implementation of the estimand framework across various guideline documents, focusing primarily on drug development for Crohn’s Disease (CD) and Uclcerative Colitis (UC), real-world studies (RWS), and pediatric extrapolation. We first discuss key principles embedded within the NMPA guidelines for drug development in CD and UC, emphasizing the treatment goal, rigorous trial designs, and considerations for estimands regarding population, variable, treatment, and intercurrent events. The chapter also illustrates the complexities of establishing estimands in RWS, highlighting considerations of diverse patient populations, treatment regimens, and intercurrent events related to behavior and nonbehavioral factors. Drawing attention to recent suggestions for pediatric extrapolation, we emphasize the necessity of clear definitions of estimands aligning with trial objectives. In closure, the chapter ascertains the importance of proactive communication between sponsors and the NMPA to ensure adherence to these guidelines and successful implementation of estimands in China.