ONCOLOGIC PHASE 0 TRIALS INCORPORATING CLINICAL PHARMACODYNAMICS: FROM CONCEPT TO THE PATIENT

摘要

The United States Food and Drug Administration recently issued an Exploratory Investigational New Drug Guidance that provides a platform for the evaluation of targeted anticancer agents in small, early phase human clinical trials that can be used to establish the feasibility of proof-of-principle target modulation assays, as well as the preliminary pharmacokinetics, and molecular imaging potential of new anticancer molecules. The exploratory IND allows for reduced requirements for manufacturing and toxicologic assessment. Early clinical trials performed in this fashion have no therapeutic intent. In this series of papers in CCR Focus, the development of this new IND mechanism, its impact on clinical trial design and clinical pharmacodynamics, the ethical implications of non-therapeutic clinical investigations, and the perspective of the pharmaceutical industry on this approach are examined.