This guidance provides information and recommendations on drug and biological product development when human efficacy studies are not ethical or feasible. The regulations that set forth the pathway for approval of these products under 21 CFR 314.600 (drugs) or 21 CFR 601.90 (biological products) are commonly referred to as the Animal Rule. 

This draft guidance revises the 2009 draft guidance for industry Animal Models – Essential Elements to Address Efficacy Under the Animal Rule. While addressing the topics covered in the 2009 draft, this revision covers a broader scope of issues for drugs developed under the Animal Rule. For example, new sections have been added related specifically to study conduct and data quality and integrity (section IV.B), development of vaccines (section VII.A), and development of cellular and gene therapies (section VII.B). There are new sections on FDA’s general expectations for animal studies related to, for example, animals used in investigations, types of 
animal care interventions, and study reports (section IV). There is also a new section on FDA’s general expectations regarding natural history studies (Appendix C). 

This guidance does not address the following topics: 

  • The chemistry, manufacturing, and controls or nonclinical pharmacology and toxicology studies necessary for drug development 

  • Issues related to initial proof-of-concept studies

  • The details of study design and conduct for drug-specific animal efficacy studies or human pharmacokinetics and/or safety studies 

  • Drug development in specific populations (e.g., children, geriatrics, and pregnant women) 

  • The development of combination products 

  • Requirements for procurement of medical countermeasures by the Federal government (e.g., Strategic National Stockpile (SNS)) 

  • The development of animal models for other purposes, such as for assessment of toxicology 


Posted on the FDA website on 31 May 2014