This chapter explores the application of the estimand framework beyond single-trial efficacy assessments, emphasizing its potential role in integrated evidence planning for drug marketing evaluations, which involves various types of clinical data from various development phases as well as real-world data. By doing so, it aims to enhance the integration of evidence across development programs and inform health authority decisions on benefits and risks. Particularly, the chapter discusses the challenges and benefits of using the estimand framework to formalize clinical questions in early-phase exploratory studies, safety evaluations, and real-world evidence studies. To this end, the chapter is structured into sections focusing on early drug development, safety and benefit-risk assessments, observational studies and real-world evidence, and comparisons between estimands and the population, intervention, control, or comparator, and outcome (PICO) framework.

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Leveraging the Estimand Framework for Integrated Evidence Planning

  • Wentian Guo,
  • Lisa V. Hampson,
  • Rima lzem,
  • Alice Wang,
  • Na Yang,
  • Qiong Du,
  • Chen Lu,
  • Jonathan M. Siegel

摘要

This chapter explores the application of the estimand framework beyond single-trial efficacy assessments, emphasizing its potential role in integrated evidence planning for drug marketing evaluations, which involves various types of clinical data from various development phases as well as real-world data. By doing so, it aims to enhance the integration of evidence across development programs and inform health authority decisions on benefits and risks. Particularly, the chapter discusses the challenges and benefits of using the estimand framework to formalize clinical questions in early-phase exploratory studies, safety evaluations, and real-world evidence studies. To this end, the chapter is structured into sections focusing on early drug development, safety and benefit-risk assessments, observational studies and real-world evidence, and comparisons between estimands and the population, intervention, control, or comparator, and outcome (PICO) framework.