January 23, 2006
The Food and Drug Administration (FDA), the government body charged with overseeing the safety and effectiveness of medications, announced new steps designed to facilitate the earliest stages of testing new drugs. The updated guidelines streamline st eps for testing small batches of experimental medicines and may speed the development of new drugs that may one day prove effective against Alzheimer's and other serious diseases.
The new guidelines apply only to the very earliest stages of drug testing. Drugs typically take years of testing, first in animals then in ever-increasing numbers of people, before they can be determined to be both safe and effective for the public at large. The process typically costs many millions of dollars and may involve thousands of experimental compounds, only a few of which ultimately become drugs sold in pharmacies. The last time a drug was approved for Alzheimer's, Namenda (also called memantine), was in 2003. Only a handful of medications are available for the memory-robbing illness.
"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," said Health and Human Services Secretary Mike Leavitt. "The recommendations announced today will help more researchers conduct earlier, more-informed studies of promising treatments so patients have more rapid access to safer and more effective drugs."
The new guidelines aim to make it easier for medical researchers and drug companies to test potential new drugs for Alzheimer's and other serious illnesses at the earliest stages. The steps will, for example, make it easier for laboratories to store materials for testing and may allow researchers to test small batches of experimental drugs in one kind of animal, rather than many, before they are tested in small numbers of people.
"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, MD, 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. Today, for the first time, medical researchers are getting specific advice from the FDA about how to safely prepare products for exploratory studies."
These early-stage exploratory studies do not show whether a drug is safe or effective in people. Rather, they may indicate which substances show potential promise and merit further study. Drug makers will still need to conduct extensive trials to show that a drug produces true benefits, safely, in people.
Exploratory research is critical to finding safe and effective new drugs for Alzheimer's disease. The Fisher Center for Alzheimer's Research Foundation is leading the way in funding such basic research in the search for a cure. To learn more, visit www.ALZinfo.org, the Alzheimer's Information Site.
Direct Final Rule: Current Good Manufacturing Practice Regulation and Investigational New Drugs
Exploratory IND Studies Federal Register
INDs Approaches to Complying with CGMP During Phase 1 Federal Register