Identification of Subgroups with Heterogeneous Treatment Effect in Dose-Response Studies

• Activity Number: 458 - New Challenges in Subgroup Analyses and Enrichment Designs
• Type: Topic Contributed
• Date/Time: Wednesday, August 2, 2017 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Bjorn Bornkamp* and Marius Thomas
• Companies: Novartis Pharma AG and Novartis
• Keywords: subgroup analysis ; dose-response ; dose-finding ; Emax model

Abstract

An important task in early phase drug development is to identify patients, which respond better or worse to an experimental treatment. While a variety of different subgroup identification methods have been developed for the situation of trials that study an experimental treatment and control (see Lipkovich et al. 2017), much less work has been done in the situation when patients were randomized to different dose groups. In this presentation I will present different options for identification of subgroups in this situation, including dose-response model based methods as well as approaches not based on assuming a dose-response model.

References Lipkovich, I., Dmitrienko, A., and B., R. D'Agostino Sr. (2017) Tutorial in biostatistics: data-driven subgroup identification and analysis in clinical trials. Statist. Med., 36: 136-196. doi: 10.1002/sim.7064.