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.
FDA. Product Development Under the Animal Rule Guidance for Industry [EB/OL]. (2015-10) [2018-04-14]. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM399217.pdf.
[2]
BEASLEY D W C, BRASEL T L, COMER J E. First vaccine approval under the FDA animal rule[J]. NP J Vaccines, 2016, 1: 16013.
[3]
FDA. Anthrax: Developing Drugs for Prophylaxis of Inhalational Anthrax Guidance for Industry.(2016-02) [2018-04-14]. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM070986.pdf.
[4]
BURNS D L. Licensure of vaccines using the animal rule[J]. Curr OpinInvirol, 2012, 2(3):353-356.
[5]
BERGMAN K L. The animal rule: the role of clinical pharmacology in determining an effective dose in humans[J]. Clin Pharmacol Therap, 2015, 98(4):365-368.
[6]
BERGMAN K L. The animal rule and emerging infections: the role of clinical pharmacology in determining an effective dose[J]. Clin Pharmacol Therap, 2009, 86(3):328-331.
[7]
BERGMAN K L, KRUDYS K, SEO S K, et al. Modeling and simulation in dose determination for biodefense products approved under the FDA animal rule[J]. J Pharmacokinet Pharmacodyn, 2017, 44(2):153-160.
[8]
YU S S, YIN H J, WANG Q L, et al. Overview of drugs approved under the Animal Rule and animal efficacy studies[J]. Chin Pharm J, 2018,53(22):71-77.
[9]
LIU L, WANG J F, LI B. Research status and progress of plague vaccine[J]. Chin Pharm J(�й�ҩѧ��־), 2013, 48(12):945-949.
2019, Vol. 54 
