DIA/FDA Biostatistics Industry and Regulator Forum

The ICH E9 (R1) Estimand Framework Guidance set the stage for constructing estimands corresponding to the clinical question of interest to be addressed by a clinical trial. Often, especially in the regulatory setting for pharmaceutical development, estimands that incorporate the totality of the patient experience are of interest. For example, a benefit-risk assessment may be required to show that the treatment's benefit outweighs the expected associated risk. Therefore, it is vital to integrate this type of assessment into the clinical question and the corresponding estimand choice. The estimand framework is particularly useful for selection of outcomes and analytic methods that reflect the benefit-risk tradeoff, as well as interpretation of intercurrent events that impact the patient experience and specification of sensitivity analyses to assess robustness of trial conclusions. In this session, leading experts in the field will discuss the estimand framework in the context of benefit-risk assessments, with illustrative examples and methodological considerations.