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65th Annual Meeting of the German Association for Medical Informatics, Biometry and Epidemiology (GMDS), Meeting of the Central European Network (CEN: German Region, Austro-Swiss Region and Polish Region) of the International Biometric Society (IBS)

06.09. - 09.09.2020, Berlin (online conference)

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Estimand in Huntington's Disease

Meeting Abstract

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  • Carrie Li - F. Hoffmann-La Roche, Basel, Switzerland
  • Giuseppe Palermo - F. Hoffmann-La Roche, Basel, Switzerland

Deutsche Gesellschaft für Medizinische Informatik, Biometrie und Epidemiologie. 65th Annual Meeting of the German Association for Medical Informatics, Biometry and Epidemiology (GMDS), Meeting of the Central European Network (CEN: German Region, Austro-Swiss Region and Polish Region) of the International Biometric Society (IBS). Berlin, 06.-09.09.2020. Düsseldorf: German Medical Science GMS Publishing House; 2021. DocAbstr. 238

doi: 10.3205/20gmds090, urn:nbn:de:0183-20gmds0905

Published: February 26, 2021

© 2021 Li et al.
This is an Open Access article distributed under the terms of the Creative Commons Attribution 4.0 License. See license information at http://creativecommons.org/licenses/by/4.0/.

Outline

Text

In this talk, we will discuss the primary estimand framework for a Phase III study in Huntington's disease, which uses a continuous endpoint as a primary outcome. While the definition of each attributes will be described, particular focus will be given to intercurrent events, including their identification and handling strategies. In addition to the primary estimation method, methods for missing data imputation and sensitivity analyses assessing the robustness of the estimator will also be discussed. Supplementary estimands to address slightly different clinical questions of interest will also be investigated. Finally we will summarize some practical considerations and challenges encountered during the process of developing the estimand for this study, including eCRF data collection and methods for imputation and estimation.

The authors declare that they have no competing interests.

The authors declare that a positive ethics committee vote has been obtained.