FDA streamlines drug testing

Agency allows molecular tests in early drug studies, helping researchers ferret out the duds.

By Aaron Smith, CNNMoney.com staff writer

January 12, 2006: 1:05 PM EST

NEW YORK (CNNMoney.com) - The Food and Drug Administration released guidelines Thursday that will help drug makers and researchers discover whether a drug compound has any tangible benefit to patients before they launch more extensive and expensive traditional tests.

FDA acting commissioner Andrew von Eschenbach, in a media teleconference, said the new system would "remove some of the hurdles in earlier phases of drug testing and development."

Nine out of 10 compounds fail in clinical studies, said Eschenbach, highlighting the hit-and-miss nature of drug testing. The new guidelines allow researchers to test drugs in small labs with limited funds before committing to large-scale tests, benefiting small, independent labs as well as major drug manufacturers, according to the FDA.

The cost of pre-clinical studies represents a "very small part" of the estimated $800 million to $1 billion spent to test each drug, said Fran Hawthorne, author of Inside the FDA. However, the savings could be massive, if the new guidelines prevent drug companies from launching money-losing tests on drugs with no benefit.

"If they catch the duds early on and don't spend the $800 million testing the drug for six years, that's where the bulk of the savings will come from," said Hawthorne, referring to the average length of time spent on testing in humans.

The FDA hopes this new system will streamline the drug review process and bring new treatments for cancer, heart disease, neurological conditions and other life-threatening disorders to the market.

In its guidelines, the FDA permits the use of new molecular imaging technology that track drug "microdoses" in pre-clinical studies. These studies could safety study, on a molecular level, the impact of drugs in small numbers of test subjects, such as seven or eight, without risks to the subjects, according to the FDA.

"Now we can learn tremendous amounts of information in exploratory study without exposing people to high doses," said Janet Woodcock, deputy commissioner for operations for the FDA.

The "microdose" pre-clinical studies would first take place in small numbers of animal subjects, and then humans, said the FDA. If the researchers find reason to continue the tests, then they would launch the traditional Phase I tests, following by Phase II and III tests. These traditional phases of testing involve hundreds or thousands of human test subjects, and the drugs are tested for safety and efficacy.

When a drug compound successfully completes Phase III, then the manufacturer typically sends a drug application to the FDA for review.

"The FDA is reforming its whole approach to manufacturing, trying to modernized manufacturing," said Woodcock. "We hope to stimulate scientific discovery. We think this will help translate new discoveries into the hands of patients."

Steven Rosenberg, chief of surgery for the National Cancer Institute, said the new guidelines will "facilitate greatly our ability to take new ideas to small numbers of patients with desperate diseases. It's going to save a lot of time in allowing us to eliminate the things that don't work in patients to those that do work."

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