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

Author

Listed:
  • Naitee Ting

    (Boehringer Ingelheim Pharmaceuticals, Inc.)

  • Lihong Huang

    (Fudan University)

  • Qiqi Deng

    (Boehringer Ingelheim Pharmaceuticals, Inc.)

  • Joseph C. Cappelleri

    (Pfizer Inc.)

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)

Suggested Citation

  • Naitee Ting & Lihong Huang & Qiqi Deng & Joseph C. Cappelleri, 2021. "Average Response over Time as Estimand: An Alternative Implementation of the While on Treatment Strategy," Statistics in Biosciences, Springer;International Chinese Statistical Association, vol. 13(3), pages 479-494, December.
    • Handle: RePEc:spr:stabio:v:13:y:2021:i:3:d:10.1007_s12561-021-09301-x
    DOI: 10.1007/s12561-021-09301-x
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    References listed on IDEAS

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    1. Melanie L. Bell & Madeleine T. King & Diane L. Fairclough, 2014. "Bias in Area Under the Curve for Longitudinal Clinical Trials With Missing Patient Reported Outcome Data," SAGE Open, , vol. 4(2), pages 21582440145, May.
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