Mol. Interv. 2007 7: 325-334.
[Summary]
[Full Text]
[PDF]
The Food and Drug Administration (FDA) recently introduced the Exploratory Investigational New Drug Guidance to expedite the clinical evaluation of new therapeutic and imaging agents. Early clinical studies performed under the auspices of this guidance, so-called "Phase 0" trials, have been initiated at the National Cancer Institute to integrate qualified pharmacodynamic biomarker assays into first-in-human cancer clinical trials. The goal of this integration is to establish proof of concept at the earliest stage of drug development. Phase 0 trials do not offer any possibility of patient benefit; instead, intensive, real-time pharmacodynamic and pharmacokinetic analyses of tumor samples and/or surrogate tissues inform subsequent trials. Phase 0 studies do not replace formal Phase I drug safety testing and require a substantial investment of resources; however, they promise more rational selection of agents for further, large-scale development as well as the identification of potential therapeutic failures early in the development process.
