Elizabeth Slate (Florida State University)
A goal of developing personalized medicine changes the focus in clinical trials to decision making for the individual, or subgroups, rather than more traditional treatment comparison via average response. This change of focus has implications for all aspects of clinical trials. The group focused on the study of the role of heterogeneity/subgroups in clinical trials in three ways: (1) prepare a review paper(s) on methods for identification of subgroups, beginning with analysis of existing clinical trials data (where a drug may have “failed” at the population level), then exploring the design of clinical trials for the purpose of identifying subgroups (“responders”), and potentially also studying methods for shrinkage over subgroups such as are used in basket trials. (2) The group studied the implications of heterogeneity/subgroups in resource allocation in clinicals, specifically the influence on decision making at interim analysis points. (3) The group attempted to quantify the contribution of a single agent in trials that evaluate sequence of agents (such as SMART studies). A 4th area of strong interest was handling multiple, competing utilities in clinical trial design, particularly incorporating patient-specific utility functions.
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