Washington Editor

WASHINGTON - A new FDA regulation allows drugs developed as antidotes to biological and chemical warfare to gain regulatory approval based on animal studies when human efficacy trials are not ethical.

Proposed in 1999, the rule goes into effect June 30 and gives the government guidelines to approve certain drugs intended to reduce or prevent serious or life-threatening illnesses that likely would not make it to market under the current rules.

The rule wasn't designed to allow companies to bypass clinical trials and quickly enter the market based on limited study, Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, told BioWorld Today. "It isn't a matter of shortening the time to market; it is more a matter of whether or not we can approve these drugs at all. Before, we couldn't approve drugs with just animal-effectiveness data."

She said drugs approved for effectiveness under this rule will be evaluated for safety under current regulations. "But we won't be able to test whether they work in people because we can't expose [healthy volunteers] deliberately to a nuclear or chemical or biological agent because it wouldn't be ethical."

However, she said a company would be required to present human efficacy data after the fact if its drug is used to treat those affected by a biological, chemical or nuclear attack.

Representatives from both the Biotechnology Industry Organization (BIO) and the Pharmaceutical Research and Manufacturers of America (PhRMA) told BioWorld Today that they support the new regulation.

"It is potentially very significant to the industry," said Steve Lawton, BIO's vice president for regulatory affairs and general counsel. "I think much of its potential lies in the response of the industry to bioterrorism, although it wouldn't be used necessarily solely for bioterrorism products. Obviously, it makes a great deal of sense in the instance of anthrax and other biological weapons."

Lawton said BIO sponsored a biodefense conference several weeks ago, "and it was just overflowing with young companies that have counterterrorism measures in the lab. To the extent that we can speed reviews of those products and extend funding, this is clearly in the national interest, and it is certainly narrow enough in scope and it has safeguards."

While the rule was proposed well before Sept. 11, Lester Crawford, FDA deputy commissioner, released a prepared statement saying, "The terrorist attacks of last fall underscored the acute need for this regulation. [This] action will help make certain that essential new pharmaceutical products are available much sooner - those products that because of the very nature of what they are designed to treat cannot be safely or ethically tested for effectiveness in humans."

But Sidney Wolfe, a spokesman for the Washington-based consumer advocacy group Public Citizen, was a little skeptical about the rule.

"The rule is obviously a good idea for all practical purposes. If you read the rule, it appears to be tightly drawn and restricted to the appropriate circumstances, but unfortunately, 10 years ago the FDA put up a regulation for fast-track drugs that were supposed to be for life-threatening circumstances that promised some therapeutic breakthrough," Wolfe told BioWorld Today. "That rule has been widely abused. A number of drugs that are not remotely a therapeutic breakthrough have been put on the fast track. The boundaries of what should have been done and what was done have been blurred and I'm very concerned the same thing might happen here."

Jeff Trewhitt, spokesman for PhRMA, countered that the industry doesn't think the fast-track designation has been abused. "All I can say is, Sidney Wolfe has never passed up an opportunity to criticize the drug approval process, the pharmaceutical industry or the FDA - and this is no different."

Wolfe said Bayer AG's Cipro was approved under a regulation that also allowed clearance without proof of efficacy.

Indeed, Woodcock said the regulation that cleared Cipro permitted approval based on a human surrogate endpoint. "That means, it didn't show that people lived. We use tests like that for AIDS. The company doesn't have to show that the drug improves survival, but they have to show that it decreases the virus," Woodcock said. "Usually, the company will do tests later [after approval] that show that the drug really improves outcome. So this would be similar - that is, if there is an event involving bioterrorism, then the company has to do follow-up to try and determine that the drug worked as expected."

The complete rule was posted in the Federal Register Friday.