Understanding molecular pathways and the identification of subgroups of patients with specific biomarker predictors of drug response related to orphan drugs

Abstract

Introduction: Thirty to forty percent of orphan drugs approved are used in the management of malignancy. This reflects the revolution in molecular oncology and biotechnology witnessed over the last 30 years. The identification of molecular pathways intrinsic to the origin of cancer has resulted in finding targets for anticancer agents and molecular biomarkers predictive of drug response. The clinical application of biomarkers in oncology has resulted in the identification of rare cancers within common cancer types. Orphan drug development plays a key role in sustaining the era of personalized cancer medicine. Areas covered: The development of crizotinib and its companion Vysis ALK Break Apart FISH Probe Kit is presented in this paper to highlight how the field of medical oncology is integrating advances in molecular biology to deliver the goal of personalized cancer care. The molecular basis of ALK-positive lung cancer and the evidence that supported crizotinib in successfully receiving an accelerated, orphan drug marketing approval status are also discussed. Expert opinion: The international oncology community strives to improve cancer outcomes. The revolution in molecular oncology has enabled the transition toward an era of targeted therapy and personalized medicine. Oncologists actively encourage pharmaceutical companies to develop novel anticancer agents in conjunction with a companion diagnostic test. This identifies a subgroup of patients most likely to respond to a drug thereby limiting unnecessary toxicity and cost. This together with the financial incentives established by the Orphan Drug Act will fuel continued research in oncology orphan drug development in association with predictive biomarkers.