Precision Cancer Medicine and Clinical Trial Design

Over the past decade, large-scale genomic surveys have uncovered a wide landscape of somatic alterations in cancers, both across different types and within histopathologically identical sub-types. The contribution of this molecular heterogeneity towards clinical outcome (e.g. overall survival, response, toxicity) has become the focus of intense study and is undoubtedly an important component of precision cancer medicine. Importantly, as our understanding of cancer as a heterogeneous and complex disease continues to evolve, so do the approaches towards therapeutic discovery [1, 2]. Currently, multiple targeted therapies to matching genomic alterations have been approved by the Food and Drug Administration (FDA) while many others are being evaluated in clinical trials. While the availability of cancer genomic testing in the clinic has led to opportunities in oncology such as drug target discovery, it has also led to challenges including how to develop targeted therapies for small populations of patients.