Abstract:
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ICH-E9(R1) has opened the door to considerations beyond ITT for clinical trials, including pivotal trials, for the primary estimand of a treatment effect. One approach that has gained some interest is the Tripartite Estimand Approach (TEA) (Akacha et al, 2017). In the TEA, one estimand is the treatment effect in the principal stratum of patients who can adhere to their study medication. Qu et al. (2020) have addressed the estimation of this estimand using causal inference approaches.
ITT is an estimand related to initiation of treatment and considers intercurrent events irrelevant to the estimation, including cessation of treatment and use of rescue medication. The TEA is aimed at the direct treatment effect - the effect of treatment when taken as prescribed. Arguments have been made that the direct treatment effect may be more relevant/understandable to patients & physicians. The impact of these estimands, estimators and estimates on product labeling will be considered. In the Roundtable, the participants will be challenged to think about different disease states or trial designs in order to compare and contrast ITT and the TEA With a guarantee of insights and learnings for all!
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