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Activity Number: 577 - Estimands: Some Perspectives from an FDA Working Group
Type: Topic Contributed
Date/Time: Thursday, August 6, 2020 : 3:00 PM to 4:50 PM
Sponsor: Biopharmaceutical Section
Abstract #312772
Title: Estimands for Use in Pulmonary-Allergy US FDA Regulatory Submissions
Author(s): Dong-Hyun Ahn* and Yongman Kim and Susan Duke
Companies: FDA and FDA and FDA
Keywords: estimands; efficacy

In regulatory interactions between the Agency and the sponsor for new drug development, agreement on estimands is beneficial because it can have a direct impact on both drug approval and labeling claims. Without prespecified estimands, there was a lack of clarity, especially with respect to how missing data would be handled and the implications on data interpretation. In 2017, ICH published an addendum to E9 Statistical Principles for Clinical Trials to provide guidance on estimands in clinical trials. However, the addendum gives a general principle on estimands, not specifically tailored to each indication. To discuss with the sponsor more effectively and efficiently, clinicians and statisticians in the Pulmonary-Allergy division derived clinically meaningful and statistically sound estimands for some common disease areas. Estimands are especially relevant to trial design, missing data handling and statistical methods. This presentation will give a brief introduction to estimands and discuss the experience in CDER’s pulmonary collaboration between clinicians and statisticians to create and use estimands we consider to be optimal for some common pulmonary diseases.

Authors who are presenting talks have a * after their name.

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