An important challenge in oncology clinical trials relates to designs to assess treatment efficacy in the context of treatments that target pathways and improved technologies to measure tumor biology. Current strategies for the evaluation of new treatments for cancer utilize designs that enrich(or target) for the population of patients who are thought to show the maximum treatment effect. We consider alternative study designs that instead target greater population impact maximizing the expected future patient outcomes both on and off the trial patients.
We show that data from Phase II trials can identify biomarker combinations and cutoffs to optimize the new objective function relating to future population impact. We then show that by conducting the follow-up phase III study using the proposed strategy, the population impact can be optimized. The proposed strategy is applicable to various types of responses, including continuous, binary and survival endpoints.
Our methods are tied to our experience in cancer our methods are tied to our experience in cancer clinical trials primarily through the SWOG Cancer Research Network and the NCI National Clinical Trials Network (NCTN).