Phase 0 clinical trials: recommendations from the Task Force on Methodology for the Development of Innovative Cancer Therapies.

The Methodology for the Development of Innovative Cancer Therapies (MDICT) task force has been established as an expert forum to develop practical guidance on the development of innovative anticancer agents, in particular targeted agents. The task force recently addressed the utility, design and application of Phase 0 clinical trials in anticancer drug development. It was concluded that the role of non-therapeutic Phase 0 trials is controversial for several reasons, including the lack of clinical benefit for participating patients. However, it was recognised that Phase 0 trials provide an opportunity to generate essential human pharmacokinetic and pharmacodynamic data earlier in the drug development process, which could be a major advantage in the design and decision making concerning further clinical development of an agent. Construction of a 'decision chart' was highly recommended to assist investigators and sponsors in determining whether an agent is suitable for evaluation in a Phase 0 trial.