In 1999, the Food and Drug Administration proposed a rule to allow the approval of drugs based solely on animal studies where human efficacy studies would be unethical. The proposal went nowhere until the Sept. 11 attacks and the anthrax scare, and the FDA announced recently that the rule would go into effect this summer.
The rules are designed to overcome an obvious problem with drugs designed as antidotes to anthrax, nerve gas, small pox and other potentially lethal or disabling agents. Who would test such drugs? Few people are likely to volunteer to be exposed to nerve gas or anthrax to test such drugs, and even if they were, such research would be widely considered unethical.
The anthrax drug Cipro was approved by the FDA based solely on animal efficacy studies for this reason, and the new FDA rules will formalize the procedures for future such drugs.
Although companies can base efficacy solely on animal studies, they will still need to show that the drug itself is safe by testing it in both animals and in human clinical trials. Such drugs will also carry warning labels noting that their efficacy has not been tested in human beings.
Sources:
FDA to OK Drugs Using Animal Data. The Associated Press, May 31, 2002.
FDA: Animal Studies ALone OK for Some Drugs. Adam Marcus, HealthScoutNews, May 30, 2002.
In Search of Antiterror Drugs. The New York Times, June 3, 2002.