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This site provides references that the working group considers relevant for the implementation of the ICH E9 addendum in drug development in pharma industry. The list is subjective and not exhaustive. If you would like to have a relevent reference added here please let us know.

References

Akacha, M., F. Bretz, and S. Ruberg. 2017. “Estimands in clinical trials - broadening the perspective.” Stat Med 36 (1): 5–19.

Bornkamp, Björn, and Georgina Bermann. 2019. “Estimating the Treatment Effect in a Subgroup Defined by an Early Post-Baseline Biomarker Measurement in Randomized Clinical Trials with Time-to-Event Endpoint.” Statistics in Biopharmaceutical Research. Taylor & Francis, 1–17.

Degtyarev, Evgeny, Yiyun Zhang, Kapildeb Sen, David Lebwohl, Mouna Akacha, Lisa V. Hampson, Bjoern Bornkamp, Antonella Maniero, Frank Bretz, and Emmanuel Zuber. 2019. “Estimands and the Patient Journey: Addressing the Right Question in Oncology Clinical Trials.” JCO Precision Oncology, no. 3: 1–10. https://doi.org/10.1200/PO.18.00381.

Hernan, M. A., and D. Scharfstein. 2018. “Cautions as Regulators Move to End Exclusive Reliance on Intention to Treat.” Ann. Intern. Med., March.

Holzhauer, B., M. Akacha, and G. Bermann. 2015. “Choice of Estimand and Analysis Methods in Diabetes Trials with Rescue Medication.” Pharm. Stat. 14 (6): 433–47.

ICH E9 working group. 2019. “ICH E9 (R1): addendum on estimands and sensitivity analysis in clinical trials to the guideline on statistical principles for clinical trials.” International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. August 2019. https://database.ich.org/sites/default/files/E9_Guideline.pdf.

Leuchs, A. K., A. Brandt, J. Zinserling, and N. Benda. 2017. “Disentangling estimands and the intention-to-treat principle.” Pharm Stat 16 (1): 12–19.

Leuchs, Ann-Kristin, Jörg Zinserling, Andreas Brandt, Dorothee Wirtz, and Norbert Benda. 2015. “Choosing Appropriate Estimands in Clinical Trials.” Therapeutic Innovation & Regulatory Science.

Magnusson, Baldur P., Heinz Schmidli, Nicolas Rouyrre, and Daniel O. Scharfstein. 2019. “Bayesian Inference for a Principal Stratum Estimand to Assess the Treatment Effect in a Subgroup Characterized by Postrandomization Event Occurrence.” Statistics in Medicine 38 (23): 4761–71. https://doi.org/10.1002/sim.8333.

Mallinckrodt, C. H., Q. Lin, I. Lipkovich, and G. Molenberghs. 2012. “A structured approach to choosing estimands and estimators in longitudinal clinical trials.” Pharm. Stat. 11 (6): 456–61.

Mehrotra, D. V., R. J. Hemmings, and E. Russek-Cohen. 2016. “Seeking harmony: estimands and sensitivity analyses for confirmatory clinical trials.” Clin Trials 13 (4): 456–58.

Rufibach, Kaspar. 2019. “Treatment Effect Quantification for Time-to-Event Endpoints – Estimands, Analysis Strategies, and Beyond.” Pharmaceutical Statistics 18: 144–64. https://doi.org/10.1002/pst.1917.

Scharfstein, Daniel O. 2019. “A Constructive Critique of the Draft Ich E9 Addendum.” Clinical Trials (London, England) 16 (4): 375–80. https://doi.org/10.1177/1740774519853566.