Phase-0: A General Overview

Development of a new drug molecule is very long and expensive operation. Out of 9000 to 10,000 compounds, one (on average) will actually reach to the market. In 2004, the US Food and Drug Administration (FDA) introduced its “Critical Path” document highlighting the serious discordance between major advances in science and technology and limited or stagnated new drug development and offer examples of newer methodologies that might be incorporated early in the drug development process to increase the efficiency of bringing a new therapeutic agent to the bedside. For this reason, the FDA issued a new Exploratory IND Guidance in 2006, to allow for such studies often called as phase 0 clinical trials which are also called as Human Micro-dosing Studies. Micro-dosing methodology is referred to as a new viable ''tool'' in the drug development toolbox. In micro-dosing studies extremely low, non-pharmacologically active doses of the drug are used to define the agent's pharmacokinetic profile in humans. In phase 0 clinical trial, animal studies can be avoided with compounds having unsuitable pharmacokinetic profiles, greatly save the investment, shorten development timeline and improve the efficiency and success of subsequent trials. The patients included in the trials are expected to be treated with better effective and more safe, efficacious drugs.