Heterogeneous treatment effects are typically explored by examining patient outcomes in mutually exclusive subgroups defined by observable patient characteristics.
For many diseases, understanding the extent and nature of treatment effect heterogeneity is key to the development of improved treatment strategies more tailored to individual patient needs. Heterogeneity of treatment effect (HTE) refers to differences in treatment effectiveness attributable to observable patient attributes such as demographic characteristics, genetic characteristics, and other baseline risk factors. However, examining only the average treatment effect in the presence of patient heterogeneity may mask important differences in treatment efficacy or in treatment safety across subsets of patients. The conventional focus of clinical trials has been on assessing the average effect of a treatment in a target population.
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