All Times EDT
Keywords: treatment policy, complex regimens, treatment sequences, rare diseases, cellular therapies, non-drug interventions
The final version of the ICH E9 (R1) addendum on estimands and sensitivity analysis in clinical trials introduced the first attribute of an estimand, the “treatment condition of interest, and, as appropriate, the alternative treatment condition to which comparison will be made”, as possibly consisting of “an overall regimen involving a complex sequence of interventions”.
This feature, which was incorporated based on feedback to the first draft released for consultation, acknowledges the potential sophistication of some clinical questions of interest. Those may identify the treatment effect of interest as the comparative overall outcome of two sets of complex and possibly interdependent interventions, delivered according to therapeutic regimens, or treatment strategies. This may involve different options, with intermediate decisions, and may lead to personalized therapeutic journeys, with the administration of all or only part of the planned interventions.
In this presentation we will review and discuss therapeutic settings in Oncology when such a definition of the “treatment” attribute may be particularly relevant for patients, prescribers, sponsors and regulators. We will discuss implications of evaluating such a “treatment strategy”, in terms of interpretation and clinical relevance of the treatment effect estimates, adequacy and efficiency of the corresponding development plans, and questions raised on the regulatory approval and labeling processes. We will contrast this approach to a development plan designed to evaluate just an investigational drug in the same therapeutic setting.