On Statistical Theory and Methodology for Targeted Clinical Trials

After completion of the Human Genome Project (HGP), the disease targets at molecular levels can be identified. As a result, treatment modality for molecular targets can be developed. In practice, targeted clinical trials are usually conducted for evaluation of the possibility and feasibility of the individualized treatment of patients. However, the accuracy of diagnostic devices for identification of such molecular targets is usually not perfect. Therefore, some of the patients enrolled in targeted clinical trials with a positive result for molecular target might not have the specific molecular targets and hence the treatment effects of the targeted therapy estimated from targeted clinical trials could be biased for the patient population truly with the molecular targets. We develop statistical methods of an unbiased inference for targeted clinical trials. The simulation studies were conducted to empirically investigate the performance of the proposed estimations and testing procedures.

Project ID:PA9907-2498
External Project ID:NSC99-2118-M182-002