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Estimands in observational studies: Some considerations beyond ICH E9 (R1).

Heng LiChenguang WangWei-Chen ChenNelson LuChanghong SongRam TiwariYunling XuLilly Q Yue
Published in: Pharmaceutical statistics (2022)
The document ICH E9 (R1) has brought much attention to the concept of estimand in the clinical trials community. ICH stands for International Conference for Harmonization. In this article, we draw attention to one facet of estimand that is not discussed in that document but is crucial in the context of observational studies, namely weighting for covariate balance. How weighting schemes are connected to estimand, or more specifically to one of its five attributes identified in ICH E9 (R1), the attribute of population, is illustrated using the Rubin Causal Model. Three estimands are examined from both theoretical and practical perspectives. Factors that may be considered in choosing among these estimands are discussed.
Keyphrases
  • clinical trial
  • working memory
  • healthcare
  • mental health
  • randomized controlled trial
  • open label
  • phase iii
  • double blind
  • placebo controlled