DIA/FDA Statistics 2015 Forum

Defining the primary objective of a clinical trial in the presence of non-compliance or non-adherence to the assigned treatment is crucial for the choice of design, the statistical analysis and the interpretation of the results. This raises the need for a structured framework to specify the primary estimand (i.e. "what is to be estimated"). The missing data report released in 2010 by the National Academy of Science, “Prevention and Treatment of Missing Data in Clinical Trials”, recommends explicit specification of a casual estimand in the protocol of a confirmatory trial. This is also reflected by the decision of ICH to amend its E9 guidance in the coming years to discuss estimands and their role in clinical trials. In this session we will discuss a clear definition of estimands and how the choice of an estimand is linked to important considerations around trial design, conduct and analysis.