Phase 0 trials: a platform for drug development?

摘要

In 2006, the US Food and Drug Administration released a guidance document outlining recommendations for exploratory investigational new-drug studies. In these so-called phase 0 studies, done before traditional phase I trials, subtherapeutic doses of a new drug are given to a small group of patients (typically fewer than 15) for roughly a week to determine pharmaco-dynamic and pharmacokinetic properties. Researchers can then use these early data to guide further development.