• Decrease font size
  • Return font size to normal
  • Increase font size
U.S. Department of Health and Human Services

Emergency Preparedness and Response

  • Print
  • Share
  • E-mail

Activities to Encourage Development, Qualification, and Use of Animal Models


Animal Rule

In May 2002, FDA issued the final rule New Drug and Biological Drug Products; Evidence Needed to Demonstrate Effectiveness of New Drugs When Human Efficacy Studies Are Not Ethical or Feasible (Federal Register notice disclaimer icon).

The regulation allows appropriate studies in animals, in certain cases, to provide substantial evidence of the effectiveness of new drug and biological products used to reduce or prevent the toxicity of chemical, biological, radiological, or nuclear (CBRN) substances.

View a high-level summary taken from the final regulation.

Animal model draft guidance

In May 2014, FDA published the revised draft guidance Product Development Under the Animal Rule (PDF, 2 MB) for comment. The revised draft provides guidance to potential sponsors (industry, academia, and government) on the development of animal models to study efficacy. FDA may rely on adequate and well-controlled animal efficacy studies to support approval of a drug or licensure of a biological product under the Animal Rule when human efficacy studies are neither ethical nor feasible. 

This revised draft guidance replaces a 2009 draft guidance for industry entitled “Animal Models--Essential Elements to Address Efficacy Under the Animal Rule.”  The revised draft addresses a broader scope of issues for products developed under the Animal Rule and incorporates the learnings of considerable product development experience.  Once finalized, this guidance is intended to help potential sponsors understand FDA’s expectations for product development under the Animal Rule.

FDA received numerous comments during the public comment period following publication of the earlier draft, and at the November 2010 public meeting on the draft guidance. To address the comments and revise the guidance, FDA created a multi-disciplinary, multi-Center team that carefully considered the comments expressed by the community, including requests to expand the scope of the guidance.

The revised and expanded guidance provides additional scientific and regulatory information to support a better understanding of the specific expectations for animal data intended to support approval or licensure of medical countermeasures.

Animal model qualification process

In January 2014, FDA published the final guidance Qualification Process for Drug Development Tools (PDF, 471 KB), which describes the qualification process for drug development tools (DDTs) intended for potential use in drug development programs.

Examples of DDTs include biomarkers, of which animal models is a subset. Both of these DDTs are important in medical product development, especially for medical countermeasures developed under the Animal Rule. Among other things, the guidance provides a framework for interactions between FDA and DDT submitters to identify the data needed to support qualification of a DDT. The guidance also creates a mechanism for formal review by FDA to qualify a DDT.

FDA is developing an Animal Model Qualification Program that will be consistent with the process described in the DDT guidance to enable the qualification of product-independent animal models within a context of use to evaluate product efficacy under the Animal Rule.

To learn more, view
Animal Rule Information or contact AskMCMi@fda.hhs.gov.