FDA rules expand human drug testing

WASHINGTON — Concerned that too few new drugs are reaching the market and that too much time is spent testing those that don't win approval, the Food and Drug Administration (FDA) on Thursday announced rules for human testing intended to make effective drugs available to the public sooner and possibly more cheaply.

Under new guidelines, investigators will be allowed to give minute doses of experimental drugs to people earlier in development to see if the results are promising enough to warrant going forward with costly, full-scale clinical testing.

Researchers and industry welcomed the FDA action. But some agency critics said they were concerned it could increase hazards for volunteers in clinical trials or facilitate the approval of drugs before their risks are fully understood.

The new system is designed to deal with a problem that has slowed getting potentially helpful medicines to patients and added billions of dollars in possibly avoidable expenses to the cost of prescription drugs. Before a new drug can be put on the market, scientists are required to prove it is effective and safe; of the thousands of compounds researchers test, few make it to the pharmacy shelf.

In the past few years, FDA officials and others have noticed a disturbing pattern: Fewer drugs made it through the required screening and many that proved unfit for the market were eliminated only near the end of the long development process. This occurs after hundreds of thousands, sometimes millions, of dollars have been spent.

Since those costs cannot be recovered from sales, drug manufacturers pass them onto consumers in the form of higher prices for drugs that do make it to the market.

The FDA's new system is designed to help scientists identify sooner the drugs that will turn out to be unmarketable.

Pharmaceutical research-and-development spending increased by about 250 percent in the past decade, approaching $39 billion last year. But the number of new drugs submitted for FDA approval declined during that period. Last year, for example, the agency approved 20 new drugs, compared with 36 in 2004.

The new FDA guidelines would enable researchers to test a "micro-dose" of an experimental drug on a small number of human volunteers.

Such testing would replace some, though not all, of the early experiments now carried out on animals.

Critics of the agency and the drug industry saw a potential danger in the new guidelines. "Last time they speeded up the process of drug approval, it led to the approval of lethal drugs," said Vera Sharav of the Alliance for Human Research Protection in New York. "Now they are trying to fiddle around with the [earliest phase of] trials? Those, by definition, are the highest-risk."

Dr. Andrew von Eschenbach, the acting FDA commissioner, said "rapid does not mean reckless" in streamlining drug development.

But Dr. Sidney Wolfe, director of Public Citizen's Health Research Group, said the proposal would save companies money at the expense of increased risk to the human subjects of trials.

"This is really a proposal — as far as I can see, an invitation — for industry to lower the costs of preclinical trials and increase the potential risk for people who will be subjects in these, quote, 'preclinical studies,' " he said.

Material from The Associated Press is included in this report.