Estimands in Real-World Studies

• Activity Number: 411 - Addressing Key Issues to Promote the Uptake of Using RWE/D for Regulatory Decisions
• Type: Topic Contributed
• Date/Time: Wednesday, August 10, 2022 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Jie Chen*
• Companies: Overland Pharmaceuticals
• Keywords: Estimands; causal inference framework; intercurrent events; observational studies; randomized controlled trials; sensitivity analysis

Abstract

Constructing the right estimand—the target of estimation—which reflects the re- search question and corresponds to the study objective, is one of the key components in formulating a clinical research. The ICH E9 (R1) describes statistical principles for constructing estimands in clinical trials with a focus on five attributes—population, treatment, endpoints, intercurrent events, and population-level summary. However, defining estimands for real-world clinical studies might require additional considerations to the ICH E9 (R1) principles, for example, due to heterogeneity of study population, complexity of treatment regimes, different types and patterns of intercurrent events, and complexities in choosing study endpoints. This paper discusses considerations in constructing extimands for real-world studies. We review similarities and differences between clinical trial and real-world study estimands and provide a roadmap for choos- ing appropriate estimands for real-world studies.