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Abstract:
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The estimand framework included in the addendum to the ICH E9 guideline facilitates discussions to ensure alignment between the key question of interest, the analysis, and interpretation. Therapeutic knowledge and drug mechanism play a crucial role in determining the strategy and defining the estimand for clinical trial designs. Clinical trials in patients with hematological malignancies often present unique challenges for trial design due to complexity of treatment options and existence of potential curative but highly risky procedures, e.g. stem cell transplant or treatment sequence across different phases (induction, consolidation, maintenance). In this talk, we illustrate how to apply the estimand framework in hematological clinical trials and how the estimand framework can address potential difficulties in trial result interpretation.
This presentation is a result of a cross-industry collaboration to connect the ICH E9 addendum concepts to applications. Real phase 3 trials will be used to consider common challenges to illustrate different scientific questions and the consequences of the estimand choice for trial design, data collection, analysis, and interpretation.
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