Drug development and clinical trials--the path to an approved cancer drug.

Abstract

Advances in our understanding of cancer biology have led to the discovery of a spectrum of new therapeutic targets. However, despite remarkable progress in the identification and characterization of novel mechanisms of the oncogenic process, the success rate for approval of oncology drugs remains low relative to other therapeutic areas. Innovative preclinical and clinical approaches, such as the use of advanced genomic technologies, as well as branched adaptive clinical trial designs, have the potential to accelerate the development and approval of highly effective oncology drugs, along with a matching diagnostic test to identify those patients most likely to benefit from the new treatment. To maximize the effectiveness of these new strategies, close collaboration between academic, industry, and regulatory agencies will be required. In this Review, we highlight new approaches in preclinical and clinical drug development that will help accelerate approval of drugs, and aim to provide more-effective treatments alongside companion diagnostic tests to ensure the right treatment is given to the right patient.