For a given estimand, an appropriate analysis method or estimator should be employed to provide an estimate that allows for a reliable interpretation. This chapter serves as a practical guide to the application of the estimand framework in analyzing continuous endpoints in clinical trials. It focuses on addressing issues arising from intercurrent events, which are the post-randomization events that may be related to treatment and outcome. We provide a detailed discussion of the treatment policy, hypothetical, and composite variable strategies for addressing intercurrent events under the estimand framework, their aligned estimators, and sensitivity analysis methods. A case study on asymptomatic Alzheimer’s disease serves to illustrate the steps of the estimand thinking process. Meanwhile, it demonstrates how different questions of interest can lead to different strategies being specified in the estimand definition for the intercurrent events of treatment discontinuation and initiation of treatment with cholinesterase inhibitors.

错误:搜索内容不能为空,请输入英文关键词
错误:关键词超出字数限制,请精简
高级检索

Statistical Analysis Under the Estimand Framework: Continuous Endpoints

  • Chen Lu,
  • Vladimir Dragalin,
  • Elena Polverejan,
  • Björn Bornkamp,
  • Marcel Wolbers,
  • Longshen Xie,
  • Na Yang

摘要

For a given estimand, an appropriate analysis method or estimator should be employed to provide an estimate that allows for a reliable interpretation. This chapter serves as a practical guide to the application of the estimand framework in analyzing continuous endpoints in clinical trials. It focuses on addressing issues arising from intercurrent events, which are the post-randomization events that may be related to treatment and outcome. We provide a detailed discussion of the treatment policy, hypothetical, and composite variable strategies for addressing intercurrent events under the estimand framework, their aligned estimators, and sensitivity analysis methods. A case study on asymptomatic Alzheimer’s disease serves to illustrate the steps of the estimand thinking process. Meanwhile, it demonstrates how different questions of interest can lead to different strategies being specified in the estimand definition for the intercurrent events of treatment discontinuation and initiation of treatment with cholinesterase inhibitors.