FDA offers guidance for efficient early-stage drug development


As part of a major new initiative to modernize current good manufacturing practice (cGMP) regulations and simplify clinical development, the FDA ( has released two documents offering guidance for advancing the earliest phases of clinical research.

Exploratory IND Studies is intended to facilitate early, exploratory studies in people, before Phase 1 safety studies begin. In the guidance, the FDA makes recommendations for safety testing, manufacturing and clinical approaches suitable for early studies.

The second guidance, INDs-Approaches to Complying with cGMP during Phase 1, outlines an approach for complying with cGMP requirements for drugs used in Phase 1 studies. In this document, the FDA recognizes specific standards for the production of the small amounts of drugs necessary for this early stage of drug development and formulates an appropriate approach to cGMP compliance.

According to Janet Woodcock, FDA Deputy Commissioner for Operations, “The problem is that researchers conducting very early studies were required to follow the same manufacturing procedures as those companies that mass-produce products for broad-scale distribution. These requirements are so burdensome for early Phase 1 studies that many leading medical research institutions have not been able to conduct these studies of discoveries made in their laboratories. Today, for the first time, medical researchers are getting specific advice from the FDA about how to safely prepare products for exploratory studies.”

Both efforts are part of the FDA’s ongoing Critical Path Initiative, launched in 2004, to make the process of bringing new drugs to market more efficient and streamlined.