Analysis on Basic Principle of Animal Rule of FDA / 中国药学杂志

ABSTRACT

OBJECTIVE: To summarize the theory about animal rule, in order to provide references for its implementation in industry. METHODS: The basic principles and main technology points of the animal rule are analyzed based on the guidance and other literatures issued by the Food and Drug Administration (FDA). RESULTS: FDA has formulated guidance, known simply as “animal rule”, which applies when clinical efficacy studies in humans cannot be ethically conducted and field efficacy studies are not feasible for certain chemical drugs and biological products including vaccines that are intended to treat or prevent serious or life-threatening diseases. Namely, those applications may be approved for marketing based on evidence of effectiveness derived from appropriate animal studies and any additional supporting data. This article introduces briefly how to carry out the critical animal effective studies and how to extrapolate human effective dose. CONCLUSION:Animal rule may provide a new registration strategy for drug candidates when their clinical efficacy studies in humans are not ethical or feasible but reasonably likely brings clinical benefits.