Scholarlink

The estimand framework has been widely applied in drug development to define trial objectives and establish clear estimands across various therapeutic areas. Unique challenges and specific considerations may arise in practice across different therapeutic areas. This chapter illustrates the practical implementation of the estimand framework in cardiovascular, respiratory, and infectious diseases and preventive vaccines through case studies in chronic heart failure (HF), asthma, coronavirus disease (COVID-19), and rabies vaccine, respectively. Each case study discusses the development of the primary estimand, strategies for addressing the intercurrent events, and the rationale behind these choices, along with the associated analysis methods. Additionally, it explores alternative estimands and relevant supplementary analyses. The thinking process illustrated in these case studies offers broad insights that are applicable to other fields.

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

Applying the Estimand Framework: Case Studies in Cardiovascular, Respiratory, and Infectious Diseases and Vaccines

  • 章节

  • 发表日期：

    2025年

查看全文
  • Alice Wang,
  • Cui Xiong,
  • Rong Fu,
  • Zhiwei Jiang,
  • Folke Folkvaljon,
  • Zelie Bailes,
  • Dawn Edwards,
  • Wenting Li,
  • Peter Mesenbrink

摘要

The estimand framework has been widely applied in drug development to define trial objectives and establish clear estimands across various therapeutic areas. Unique challenges and specific considerations may arise in practice across different therapeutic areas. This chapter illustrates the practical implementation of the estimand framework in cardiovascular, respiratory, and infectious diseases and preventive vaccines through case studies in chronic heart failure (HF), asthma, coronavirus disease (COVID-19), and rabies vaccine, respectively. Each case study discusses the development of the primary estimand, strategies for addressing the intercurrent events, and the rationale behind these choices, along with the associated analysis methods. Additionally, it explores alternative estimands and relevant supplementary analyses. The thinking process illustrated in these case studies offers broad insights that are applicable to other fields.