All Times EDT
Keywords: precision medicine, bridging study, CDx, patient enrollment
Precision medicine, a medical model that separates patients into different subpopulations with medical decisions, practices, interventions, and/or products, is becoming increasingly prominent. Particularly, during the drug-diagnostic device co-development, genomic diagnostics can be used to identify patients who might benefit from investigational, often cutting-edge therapies, while sparing those who do not have a particular genetic signature from the costs and side effects of certain treatments. Other than a well-characterized market-ready companion diagnostic (CDx) assay, usually a clinical trial assay (CTA) is used for patient enrollment in the drug pivotal clinical trial. Consequently, a bridging study needs to be conducted to estimate the efficacy of the drug in the population defined by CDx. However, in practice, the trial enrollment procedure may be much complicated, for example, using multiple laboratory developed tests (LDTs) to accelerate the patient enrollment. In this presentation, I will discuss practical issues and statistical challenges in complex bridging study designs, and a case trial involving both tissue and liquid based assays will be demonstrated.