Animal efficacy rule

A regulatory pathway for specific indications in the United States of America.

When human efficacy studies are not ethical or practicable, the US Food and Drug Administration (FDA) may grant approval based on well-controlled animal studies, when the results of those studies establish that the drug or biologic product is reasonably likely to produce clinical benefit in humans. The product sponsor must still demonstrate the product’s safety in humans. Example: drugs for potential bioterror threats.

See also: Animal model, efficacy, Neutropenic mouse model