Phase 0: A New Era in Drug Development

In a nutshell new guidance for Investigational New Drug applications will give the slow and methodical drug development process a much needed shot in the arm, favoring early stage life science companies and academia.

The FDA has released new guidelines designed to allow entrance into human Phase 0/I clinical trails with human microdosing based upon rat data. The new standards also cover the manufacturing process of these drug substances via the reduction of certain cGMP standards; an extensive and expensive set of standards often difficult for early stage companies to achieve.

Researchers may perform these new studies using microdoses of drugs in human subjects before the completion of traditional pre-clinical studies. A microdose is equal to 1/100th of the amount of dose at which a drug would be expected to have therapeutic effect. The implementation of human microdose studies enables certain pharmacokinetic and bioavailability data to be quickly and inexpensively derived thus allowing improved compound selection and reduced attrition rates in early clinical development. Ultimately this practice will enable drug developers to better identify and optimize these early stage compounds prior to the advancement into full-scale clinical trials, thereby to a certain extent mitigate the inherent high risk and high cost process of bringing a drug candidate through the approval process and to market.

Here are some resources for more in-depth information:

1) INDs — Approaches to Complying with CGMP During Phase I

2) Improved Early Clinical Development Through Human Microdosing Studies

3) Human Microdosing: A Practical Guide