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Overview | For product sponsors | Compliance program for nonclinical labs | Data standards | Animal Rule links | Product approval information


Before a medical product can be approved by FDA, the sponsor must prove efficacy—that the product works. In some cases, such as developing medical countermeasures (MCMs) for potential bioterror threats, human challenge studies (exposing people to the threat agent) would not be ethical or feasible.

In these cases, FDA may grant approval based on well-controlled animal studies, when the results of those studies establish that the drug or biologic product is reasonably likely to produce clinical benefit in humans. The product sponsor must still demonstrate the product’s safety in humans.

For product sponsors

FDA strongly encourages MCM sponsors to establish early and ongoing communications with FDA. 

The Pandemic and All-Hazards Preparedness Reauthorization Act of 2013 (PAHPRA) added a new section 565(d) of the FD&C Act to require that FDA establish a procedure for a sponsor or applicant developing “a countermeasure for which human efficacy studies are not ethical or practicable, and that has an approved investigational new drug application or investigational device exemption” (IND and IDE, respectively) to request and receive two meetings with FDA – one meeting to discuss “proposed animal model development activities” and a second meeting prior to initiating pivotal animal studies. More MCM-related counterterrorism legislation

Drug products and biologics

Guidance for Industry: Product Development Under the Animal Rule (PDF, 574 KB)

FDA intends to rely on its existing procedures for arranging formal meetings with sponsors and applicants to enable them to request and receive the meetings provided in section 565(d).  Thus, sponsors or applicants developing drug products should consult the following resources for detailed information about the process and expectations for meetings about product development under the Animal Rule, in addition to the guidance linked above:

  1. Guidance for Industry - Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products (PDF, 156 KB) (December 2017)
  2. FDA’s Drug Development Tools Qualification Programs

Compliance Program for the Inspection of Nonclinical Laboratories Conducting Animal Rule-Specific Studies

On April 1, 2019, FDA posted a Compliance Program for the Inspection of Nonclinical Laboratories Conducting Animal Rule-Specific Studies (CP 7348.007) (PDF, 173 KB) on its Bioresearch Monitoring Program (BIMO) Compliance Programs web page.

This compliance program provides instructions for the inspection of nonclinical laboratories conducting the Animal Rule-specific studies (i.e., the natural history studies that define the animal model in which the efficacy of an investigational drug or biological product will be tested, the adequate and well-controlled animal efficacy studies intended to provide the primary evidence of effectiveness to support marketing approval of the product, and the pharmacokinetic and/or pharmacodynamic studies in animals used to select a dose and regimen in humans).

Inspections of these studies are conducted to verify, to the extent practicable, the quality and integrity of the data contained in the final reports of the Animal Rule-specific studies submitted to FDA.

Data Standards for Animal Rule Studies

FDA worked with the Clinical Data Interchange Standards Consortium (CDISC) to develop electronic data standards for the natural history and efficacy studies conducted in animals that support Animal Rule applications. The Standard for Exchange of Nonclinical Data (SEND) Implementation Guide-Animal Rule v1.0 (SENDIG-AR v1.0) was published by CDISC on September 17, 2019, and FDA’s support for these data standards began on March 15, 2020.

SEND data sets are required in Animal Rule submissions to the Center for Drug Evaluation and Research (CDER) for studies initiated after March 15, 2022, for new drug applications (NDAs), abbreviated new drug applications (ANDAs), and biologics license applications (BLAs) and for studies initiated after March 15, 2023, for certain investigational new drug applications (INDs).

Animal Rule links

Product approval information

Related links


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