Average Response over Time as Estimand: An Alternative Implementation of the While on Treatment Strategy

Naitee Ting, Lihong Huang (), Qiqi Deng and Joseph C. Cappelleri
Additional contact information
Naitee Ting: Boehringer Ingelheim Pharmaceuticals, Inc.
Lihong Huang: Fudan University
Qiqi Deng: Boehringer Ingelheim Pharmaceuticals, Inc.
Joseph C. Cappelleri: Pfizer Inc.

Statistics in Biosciences, 2021, vol. 13, issue 3, No 6, 479-494

Abstract: Abstract Missing data in longitudinal analysis has long been an important concern for drug developers and regulators. ICH published its E9 (R1) guidance in 2019, addressing the issue of estimands. In that guidance, five strategies are introduced. This manuscript focuses on the While on Treatment strategy, and attempts to propose average response over time as an alternative to last observation carried forward in the application of While on Treatment strategy. This is a simple summary statistics reflecting the average change a patient experiences during the trial. It is easy to interpret and easy to understand by non-statisticians. In clinical trials, with longitudinal data, average response over time has many applications. Two estimators are proposed to implement this estimand—simple mean over time, and standardized area under the curve (sAUC)

Keywords: Estimand; Last observation carried forward; Average response over time; Standardized area under the curve; Simple mean over time; Analysis of covariane; While on treatment strategy (search for similar items in EconPapers)
Date: 2021
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DOI: 10.1007/s12561-021-09301-x

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