New FDA guidance allows small studies in humans before phase I safety testing.(News)(investigational new drug studies)

Researchers now have a pathway for conducting early clinical testing of drugs in a small number of human subjects under new guidance from the Food and Drug Administration.

Officials at the FDA finalized guidance on exploratory investigational new drug (IND) studies which allows researchers to move forward with small human studies before beginning traditional phase I safety testing in humans. The guidance, published in January, makes recommendations on safety testing, manufacturing, and clinical approaches in these early studies.

The FDA also published draft guidance and a direct final rule last month that outlines new standards for the manufacture of drugs solely for use in phase I studies. The rule is aimed at making it easier for scientists to produce small quantities of drugs for small-scale, early-phase human testing.

"This is about saving lives and about building medicine's future," said Dr. Andrew von Eschenbach, acting FDA Commissioner of Food and Drugs.

Currently, less than 10% of IND applications for new molecular entities progress beyond the investigational stage, according to the FDA. These changes will remove some of the hurdles from very early drug development, Dr. von Eschenbach said during a media teleconference sponsored by the FDA.

But critics of the approach say it relaxes needed human subjects protections at a time when the safety of clinical trials is already being questioned.

In guidance on the exploratory IND, FDA officials outline their thinking that drug sponsors have not taken full advantage of the flexibility in the existing regulations and often provide more supporting information than is required for an exploratory IND.