Realizing the benefits of the estimand framework when reporting and communicating clinical trial results-some recommendations.

Abstract

Reports of the results of clinical studies are integral to regulatory decision making. They are used to support marketing authorization, to substantiate labeling information, and to inform academic publications, trial postings, and promotional messages intended to communicate study results to doctors and patients. Therefore, beyond summarizing the study design, methods, and data, study reports should provide clear descriptions of the benefits and risks of an intervention for a given medical condition. The ICH E9(R1) addendum on estimands and sensitivity analysis in clinical trials provides a framework aiming for more clarity about the reported "treatment effects." In practice, stakeholders are still learning how to embrace the estimand framework and how it impacts the reporting of study results in the most value-adding manner. This paper provides recommendations and considerations for implementing the estimand framework in the reporting of results to realize its full potential of increased transparency for interpretation and decision-making. These recommendations are based on practical experiences of working with clinical trial teams through the reporting process following implementation of the estimand framework in the protocol. As illustrated by two case studies, the primary application is in reporting studies for the scientific evaluation of medicines by regulatory agencies, directly impacting clinical study reports and submission documents, and will also extend to publications in scientific journals.