PURPOSE: The EMA recently adopted the ICH E9 R1 document on estimands. We discuss how to apply the estimand framework across different aspects of outcomes research, including PROs and RWE. Participants will become familiar with estimands and be able to critically evaluate and apply strategies for constructing estimands for different endpoints.

DESCRIPTION: The newly approved ICH E9 R1 document on estimands is an effort to align trial objectives, design and analysis, improve communication, and clarify interpretation of the effect of treatment. As ICH member organizations adopt the document, understanding and constructing estimands will be critical going forward. This workshop will cover a) how concepts from causal inference can help with understanding estimands, b) designing and communicating an estimand strategy for PRO endpoints in challenging trial settings, c) a regulatory perspective on key considerations for estimands, and d) how the estimand framework can be applied for RWE.

Dr. Gutman will discuss the potential outcomes framework for causal inference and show how it relates to estimands and can facilitate the interpretation of treatment effects. Dr. Roydhouse will provide an example using estimands for PRO endpoints in trials with seriously ill patients and discuss strategies for PRO analysis and communicating PRO results. Dr. Mishra-Kalyani will describe regulatory thinking regarding estimands and considerations such as treatment discontinuation. Dr. Manson will discuss the challenges for trial planning and communication using estimands in RWE and HEOR, and strategies for trial design and analysis that incorporate aspects such as crossover and immature data for secondary endpoints (e.g. survival).

Each presentation will be 10 minutes to allow time for discussion. Online polling will be used encourage audiences to share their experiences and questions. The workshop will be beneficial to outcomes researchers and industry and patient stakeholders interested in understanding recent developments in regulatory evaluation, trial design, PROs and RWE.