Collaboratively Improve Trial Design and Result Interpretation Through the Estimand Framework

• Activity Number: 161 - Collaboratively Improve Trial Design and Result Interpretation Through the Estimand Framework
• Type: Topic-Contributed
• Date/Time: Tuesday, August 10, 2021 : 10:00 AM to 11:50 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Paul Kluetz* and Gregory Levin* and Bohdana Ratitch* and Anthony Man* and Sally Seymour*
• Companies: The US FDA and The US FDA and Bayer and Novartis and Food and Drug Administration
• Keywords: estimand ; estimand strategy; intercurrent events; clincal question of interest

Abstract

The implementation of the ICH E9(R1) guideline requires close collaboration between clinicians and statisticians, using a common language under the estimand framework. In this session, experts from academia, industry, and regulatory bodies will discuss the impact and implementation of this new ICH guideline. The questions to the panel are: 1. What is the estimand framework? Is the framework an attempt to repackage old wine in new barrels? What exactly are we expected to do differently tomorrow that we are doing well enough today? 2. How do we handle disagreements about choosing different strategies and different population-level statistics, especially when the choice may depend on policy and regulatory considerations? 3. Has the estimand framework achieved its goal to improve the design, conduct, and analysis of clinical trials? If not, where is improvement needed? . ICH E9(R1) mainly focuses on efficacy for randomized studies. What is the role of estimands in safety evaluations, risk-benefit analyses, and observational studies? 5. Has the estimand framework been adopted by the medical community? If not, which areas need to be improved?