The US Food and Drug Administration (FDA) issued new guidelines to advance the earliest phases of clinical research in the development of innovative medical treatments. FDA's goal is to improve the process for bringing safe and effective drugs for potentially serious and life-threatening diseases, such as cancer, heart disease and neurological disorders, to the market.
The guidelines suggest specific approaches for researchers who are planning to conduct very early clinical studies in people and offer approaches for performing appropriate safety testing and producing small amounts of drugs safely. In line with the aims of FDA's Critical Path Initiative to modernise the drug development process, these changes will enable US medical researchers to evaluate much more efficiently the promise of scientific advances discovered in their laboratories, claims a FDA release.
"Currently, nine out of ten experimental drugs fail in clinical studies because we cannot accurately predict how they will behave in people based on laboratory and animal studies. These recommendations will help more researchers conduct earlier, more-informed studies of promising treatments so patients have more rapid access to safer and more effective drugs," said Health and Human Services secretary Mike Leavitt.
The FDA also outlines a suggested approach to complying with current good manufacturing practice (cGMP) requirements for drugs intended for use solely in phase 1 studies. With this new guidance and an accompanying regulation, FDA formally recognises specific standards for the manufacture of small amounts of drug product for phase 1 studies and formulating an approach to cGMP compliance that is appropriate for the particular stage of drug development.
"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. For the first time, medical researchers are getting specific advice from the FDA about how to safely prepare products for exploratory studies," said Janet Woodcock, FDA deputy commissioner for operations.
These efforts are part of the agency's Critical Path Initiative, launched in a March 2004. The goal of the Critical Path Initiative is to reduce the time and resources expended on candidate products that are unlikely to succeed, by creating new tools to distinguish earlier in the process those candidates that hold promise.