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Activity Number: 113
Type: Topic Contributed
Date/Time: Monday, August 4, 2014 : 8:30 AM to 10:20 AM
Sponsor: Biopharmaceutical Section
Abstract #313152 View Presentation
Title: Selection of Development Pathways with Established or Exploratory Biomarkers
Author(s): Ming-Xiu Hu*+ and Yi Liu and Feng Gao
Companies: Takeda and Takeda and Takeda
Keywords: Personalized Medicine ; Biomarker ; Innovative Design ; Type I Error Control ; Go/No Go Decision Rule ; Return on Investment
Abstract:

As personalized medicine becomes a key theme in drug development, selection of patient populations based on biomarkers becomes an important part of drug development decision making. When a biomarker is fully established in a disease setting, the decision is to choose between the whole population and some biomarker-defined subpopulations or to incorporate both in the trial. If the biomarker has not been fully established, many design options and development pathways will have to be evaluated and considered. Under the latter setting, type I error control can be a challenging issue. Problems have been identified with methods published in the literature and new methodologies are needed when the selection of the biomarker or its threshold is part of the trial objectives. This presentation will discuss how statisticians may influence the decision making process and the decision itself in both pivotal and non-pivotal trials. We will describe a simulation tool for assisting the development team and the company management to make better decisions. A few innovative designs and their associated statistical methods will be discussed and real examples will be used to illustrate the points.


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