FDA Guidances for Early Human Testing of New Drugs

BETHESDA, Maryland—Officials at the National Cancer Institute have welcomed two new guidance documents issued by the FDA. The two aim at making it easier for clinical researchers to conduct small-scale human studies of exploratory drugs prior to phase I trials. The documents are designed to increase the number of promising drugs that researchers can evaluate by administering them at microdose levels to small numbers of patients before deciding whether the agents warrant further human study.

The Exploratory IND Studies guidance addresses safety tests, manufacturing, and clinical approaches to conducting very early human testing to determine a drug's distribution and activity in the human body. It advises researchers how they can take advantage of the flexibility that exists in FDA regulations when they seek the agency's approval to proceed with such a study.

The second guideline describes an approach researchers can take that will satisfy the agency's Current Good Manufacturing Practice (CGMP) requirements related to drugs intended solely for use in phase I studies. This guidance will enable researchers to evaluate investigational drugs without expending time and money to show they have the capacity to produce the drugs on a large scale.

According to FDA, the new guidances will enable researchers to more efficiently evaluate drug discoveries made in their own laboratories. "The problem is that researchers conducting very early studies were required to follow the same manufacturing procedures as those companies that mass produce products," said Jane Woodcock, MD, FDA's deputy commissioner for operations. "These requirements are so burdensome for early phase I studies that many leading medical research institutions have not been able to conduct these studies of discoveries made in their laboratories."