|
Abstract:
|
Applications of personalized medicine are becoming increasingly prominent. A well-characterized market-ready companion diagnostic assay (CDx) is often desired for patient enrollment in device drug pivotal clinical trial(s) so that Food and Drug Administration can ensure that appropriate clinical and analytical validation studies are planned and carried out for CDx. However, such a requirement may be difficult or impractical to accomplish. A clinical trial assay (CTA) instead of CDx may be used for patient enrollment in the clinical trial. However, during the course of clinical trial, CDx may be available as such CTA will be switched to CDx as the clinical trial enrollment assay. A so-called partial bridging study is needed to assess the agreement between CDx and CTA in order to bridge the clinical data from CTA to CDx. In this paper, we will discuss statistical challenges in study design and data analysis for the partial bridging study. Particularly, we aimed to provide statistical methods on how to estimate the drug efficacy in CDx intended use population using results from the partial bridging study and pivotal clinical trial.
|
