The Precision Medicine Initiative is an NCI driven program in cancer to generate the scientific evidence needed to move the concept of precision medicine into clinical practice. The rapid development and widespread availability of next generation sequencing and other molecular interrogation of tumors has heralded a new era of knowledge about each individual’s tumor at a point in time. In some instances, this information has led to new therapeutic discoveries, in most instances, this information has been uninformative or of unclear significance. The NCI Molecular Analysis for Therapy Choice (MATCH) trial [NCT02465060] which screens for molecular features that may predict response to a drug with a given mechanism of action, is a multi-study, collaborative effort between the NCI and many pharmaceutical companies to begin to clarify the significance of molecular alterations in tumors not previously studied. This trial design is in response to the recent appreciation that certain driver mutations which may be common in a particular tumor type are mutated in other diseases at low frequency (<10%). In low frequency mutations, testing the utility of certain targeted therapy requires screening large numbers of patients. This review article will discuss the types of novel trial designs that led to the development and launch of the NCI MATCH.
Copyright © 2015. Published by Elsevier Inc.