Volume 27, Issue 4 - Patient-reported outcomes

Estimands – closing the gap between study design and analysis

Authors: Helen Bridge, Thomas M. Schindler

Abstract

Download the full article

References

1. International Council for Harmonisation of Technical Requirements for Pharma ceuti - cals for Human Use [Internet]. Estimands and sensitivity analysis in clinical trials. E9 (R1). Current Step 2 version. 2017 Jun 16 [cited 2018 Aug 19]. Available from: Available from: http://www.ich.org/fileadmin/Public_ Web_Site/ICH_Products/Guidelines/ Efficacy/E9/E9-R1EWG_Step2_ Guideline_ 2017_0616.pdf.
2. European Medicines Agency [Internet]. Overview of comments received on draft ICH E9(R1) addendum on estimands and sensitivity analysis in clinical trials to the guideline on statistical principles for clinical trials. EMA/744060/2017. 2017 Nov 09 [cited 2018 Aug 19]. Available from: Available from: http://www.ema.europa. eu/docs/en_GB/document_library/Over view_of_comments/2018/04/WC500247 968.pdf.
3. ICH E9(R1) Expert Working Group [Internet]. Draft (Step 2) guideline ICH E9(R1) Estimands and Sensitivity Analysis in Clinical Trials (training material). 2018 Jun [cited 2018 Oct 21]. Available from: Available from: https://www.ich.org/fileadmin/ Public_Web_Site/ICH_Products/ Guidelines/Efficacy/E9/E9-R1EWG_ Modules1–3_Step2_COMPILATION _Training Material_2018_0703.pdf.
4. Hemmings R, Akacha M [Internet]. ICH E9 (R1) Addendum on ‘Estimands and Sensitivity Analysis’. Presentation at the Annual Conference of the International Society of Clinical Biostatistics 2017 – Mini Symposia. 2017 [cited 2018 Aug 19]. Available from: Available from: http://iscb2017.info/ uploadedFiles/ISCB2017.y23bw/file Manager/09%2045%20ROB%20 HEMMINGS%20MOUNA%20AKACHA %20 Estimands%20Vigo%20 Final.pdf.
5. Akacha M, Bretz F, Ruberg S. Estimands in clinical trials – broadening the perspective. Stat Med. 2017;36:5–19. Available from: http://
6. Ruberg SJ, Akacha M. Considerations for evaluating treatment effects from randomized clinical trials. Clin Pharmacol Ther. 2017;102:917–23. Available from: http://
7. Akacha M, Kothny W. Estimands: a more strategic approach to study design and analysis. Clin Pharmacol Ther. 2018;102:894–96. Available from: http://
8. Mallinckrodt C, Molenberghs G, Rathmann S. Choosing estimands in clinical trials with missing data. Pharm Stat. 2017;16:29–36. Available from: http://
9. Phillips A, Abellan-Andres J, Soren A, et al. Estimands: discussion points from the PSI estimands and sensitivity expert group. Pharm Stat. 2017;16:6–11. Available from: http://
10. Holzhauer B, Akacha M, Bermann G. Choice of estimand and analysis methods in diabetes trials with rescue medication. Pharm Stat. 2015;14:433–47. Available from: http://
11. Leuchs A-K, Zinserling J, Brandt A, et al. Choosing appropriate estimands in clinical trials. Ther Innov Regul Sci. 2015;49: 584–92. Available from: http://
12. Food and Drug Administration [Internet]. Presentations for the July 19, 2011 meeting of the Endocrinologic and Metabolic Drugs Advisory Committee. 2011 [cited 2018 Aug 19]. Available from: Available from: https://wayback.archive-it.org/7993/ 20170405220520/https://www.fda.gov/ downloads/AdvisoryCommittees/ CommitteesMeetingMaterials/Drugs/ EndocrinologicandMetabolicDrugs AdvisoryCommittee/UCM264312. pdf
13. Bridge H, Schindler TM. The perils of the unknown: missing data in clinical studies. Med Writ. 2018;27:56–9. Available from: http://