Interplay Between Estimands and Missing Data in Clinical Trials

• Activity Number: 551 - New Innovations in Handling Incomplete Biomedical Data in the Era of Data Science
• Type: Invited
• Date/Time: Wednesday, August 2, 2017 : 2:00 PM to 3:50 PM
• Sponsor: International Indian Statistical Association
• Author(s): David Ohlssen*
• Companies: Novartis
• Keywords: Missing data ; Estimand ; Sensitivity analysis ; Clinical trials ; Drug development

Abstract

The National Research Council (NRC) highlighted the need to more clearly distinguish between the target of estimation ('estimand') and the method of estimation ('estimator') in clinical trials. While the NRC report on "The Prevention and Treatment of Missing Data in Clinical Trials" focuses on issues arising due to missing data, a framework to coherently align trial objectives and corresponding estimands is valuable more generally. TheInternational Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) has reinforced this by tasking a working group to develop an addendum to the ICH-E9 guideline "Statistical Principles for Clinical Trials". In this talk we motivate the need for change, propose a structured framework to bridge trial objectives with proper inference tools and discuss how it may impact the role of statisticians involved in clinical trial design and analysis. We illustrate these ideas using a number of examples from a drug development setting.