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Session 10: The Estimand Framework in the Context of Benefit-Risk Assessments
Session Chair(s)
Guoxing Soon, PHD
Lead Mathematical Statistician, FDA, United States
Lisa Wruck, PHD
Associate Professor of Biostatistics and Bioinformatics, Duke School of Medicine, United States
The ICH E9 (R1) Estimand Framework Guidance set the stage for constructing estimands corresponding to the clinical question of interest to be addressed by a clinical trial. Often, especially in the regulatory setting for pharmaceutical development, estimands that incorporate the totality of the patient experience are of interest. For example, a benefit-risk assessment may be required to show that the treatment's benefit outweighs the expected associated risk. Therefore, it is vital to integrate this type of assessment into the clinical question and the corresponding estimand choice. The estimand framework is particularly useful for selection of outcomes and analytic methods that reflect the benefit-risk tradeoff, as well as interpretation of intercurrent events that impact the patient experience and specification of sensitivity analyses to assess robustness of trial conclusions. In this session, leading experts in the field will discuss the estimand framework in the context of benefit-risk assessments, with illustrative examples and methodological considerations.
Learning Objective : - Identify the components of the estimand framework in the context of benefit-risk assessments
- Describe strengths of the estimand framework for jointly assessing efficacy and safety of a treatment and accounting for intercurrent events that also impact the patient experience
- Identify challenges in assessing impact of a treatment on the total patient experience and describe methods to address these challenges
Speaker(s)
Gregory Levin, PHD
Associate Director for Statistical Science and Policy, OB, OTS, CDER, FDA, United States
Speaker
Frank Bretz, PHD
Distinguished Quantitative Research Scientist, Novartis , Switzerland
Speaker
Scott Evans, PHD, MS
Professor and Founding Chair, Department of Biostatistics and Bioinformatics, George Washington University Milken Institute of Public Health, United States
Speaker
Stephen Ruberg, PHD, MS
President; Adjunct Professor of Statistics, Purdue University, Analytix Thinking, United States
Speaker
Lei Nie, PHD
Director, Division of Biometrics IV, Office of Biostatistics, OTS, CDER, FDA, United States
Speaker
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