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.
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 of the final regulation
- More Animal Rule information, including product approvals
Animal model guidance
Final Guidance for Industry: Product Development Under the Animal Rule (PDF, 574 KB)
FDA published the revised draft guidance "Product Development Under the Animal Rule" for comment in May 2014, and published the revised final guidance in October 2015. This document 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This revised guidance replaces a 2009 draft guidance for industry entitled “Animal Models--Essential Elements to Address Efficacy Under the Animal Rule.” The revised final guidance addresses a broader scope of issues for products developed under the Animal Rule and incorporates the learnings of considerable product development experience. 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the public comment period following publication of the earlier draft, and at the November 2010 public meeting on the draft guidance.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
Final Guidance for Industry: Qualification Process for Drug Development Tools
In January 2014, FDA published the final guidance Qualification Process for Drug Development Tools, 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.