The incorporation of biomarkers in drug development is widely viewed as a fundamental component to the success of clinical development programs, and in the pursuit of finding the “right” biomarker we are often asked to deliver biomarker assays with little information on the intended use of the data, but rather the notion of “develop the best assay as possible.” The reality is that what may be the best assay for a given context of use (COU) may not be appropriate for another COU. With each iteration of the COU, the biomarker assay and associated validation data must evolve to be fit-for-purpose. Alternatively, we may conclude that there are limits on the possible context of uses for a biomarker. Here we present a case study that shows the data-driven and stage-gated approach to developing biomarker assays as the COU evolves from preliminary exploration of a biomarker of interest, to guiding clinical development decisions, and ultimately to monitor patients in the real world.
Understand how to iterate an assay to meet evolving COUs
Determine key considerations for COU-driven assay characterization and validation and evaluate existing assays/developing assays for their suitability for proposed COUs
Educating stakeholders how to define COU and craft specific COU statements to support drug development programs at all stages