The new standards have been welcomed by US pharmaceutical researchers and manufacturers and will be particularly beneficial for smaller companies and academia who currently struggle with the strict current good manufacturing practice (cGMP) requirements demanded by the FDA for drugs entering Phase 1 clinical trials.
"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," said Janet Woodcock, FDA deputy commissioner for operations.
"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," she said.
The guidance relaxation is good news for the flagging drug discovery industry, as one of the biggest barriers research and academic institutions face is the ability to get discoveries made in the lab into clinical testing to begin with.
As a symptom of this, the success rate of a new molecule being developed into a marketable compound is now as low as 5-10 per cent and the overall cost of bringing a new drug to market appears to be increasing at a rate of nearly 50 per cent every 5 to 7 years, according to a recent study at Purdue University.
Much of a new drug's clinical development is held back by the drug supply, as once a compound has been successfully developed, the manufacturing process can be extremely variable.
Because of this, clinical trials are often delayed while the company waits for enough of the trial drug needed to dose all patients for the entire trial to be manufactured according to cGMP standards.
This can be a costly and timely process, therefore the new relaxation of manufacturing rules for making compounds for early studies could lead to a dramatic reduction in time and resources required to begin these early clinical studies.
The guidance relaxation only applies to Phase 1 studies, which include initial studies to determine the metabolism and pharmacological actions of drugs in humans, the side effects associated with increasing doses, and to gain early evidence of effectiveness. At this stage, drugs are tested in a small group of healthy volunteers to determine the drug's activity.
The move is part of FDA's new commitment to modernise existing cGMP regulations to streamline clinical development and is part of the Agency's Critical Path Initiative, launched in March 2004, with the goal of reducing 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.
It is hoped that this will 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.
In the new guidance and an accompanying regulation, the FDA outlines a suggested approach to complying with cGMP requirements for drugs intended for use solely in phase 1 studies.
In addition, the FDA formally recognizes 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.
"For the first time, medical researchers are getting specific advice from the FDA about how to safely prepare products for exploratory studies," said Woodcock.
To view the new guidance, go to the FDA >website.