Keywords: safety estimands, clinical trial design, clinical trial data interpretation
Room: Madison B
Recent guidance on the need for increased specificity of clinical trial objectives has driven a widespread adoption of refined targets of estimation. This increased specificity, in conjunction with pre-specified strategies for handling intercurrent events, significantly affects trial design and the interpretation of clinical trial results. While the implementation of the estimand framework has been largely confined to the assessment of efficacy and effectiveness, regulatory preferences suggests that a similar approach for the assessment of safety is also warranted. This roundtable will discuss fundamental issues that may arise in implementing safety estimands in clinical trials.
Q1. Would the adoption of such a framework likely improve patient safety, either in clinical trials or following the approval of an investigational compound? Q2. Mallinckrodt, et al. outlines 6 estimands for consideration for efficacy/effectiveness; what estimands would you consider as appropriate for safety endpoints? Q3. What are possible implications on trial design and data interpretation as it relates to regulatory preference (e.g. ITT estimands versus per-protocol estimands).
Mallinckrodt, C.H., Lin, Q. Lipkovich, I. and Molenberghs, G. (2012). A structured approach to choosing estimands and estimators in longitudinal trials. Pharmaceutical Statistics, 11, 456-461