|
Abstract:
|
Traditional randomized clinical trials (RCTs), which focus on the average treatment effect in the overall population by testing the treatment in an unselected or untargeted population (i.e. all-comer design). However, many new anticancer agents are molecularly targeted and might only benefit a subgroup of patients. Efficient development of new anticancer drugs might, therefore, need to use clinical trial designs that take into account response heterogeneity between different subgroups. We proposed an improved biomarker-guided adaptive patient enrichment design for oncology trials in which both early efficacy and futility are allowed. Sample size re-estimation could be performed to improve study power for both overall population and marker-positive group. More logical and sensible enrichment criteria has been implemented for interim enrichment decision making. In addition, different testing procedures to control family-wise type I error rate and its impact on study power have been evaluated as well.
|
