Animal Model Qualification: Frequently Asked Questions (FAQs)
What types of animal models could be considered for qualification by the Animal Model Qualification Program?
The qualification program will consider animal models of human diseases or conditions caused by chemical, biological, radiological, or nuclear (CBRN) substances for which product development would require use of the regulations commonly known as the Animal Rule. The Animal Rule can be used for drugs and biological products intended to ameliorate or prevent serious or life-threatening conditions due to toxic CBRN substances (e.g., organophosphorus nerve agent, smallpox virus, gamma irradiation) when it would not be ethical to conduct human challenge studies and field trials to study effectiveness are not feasible and when the proposed investigational product cannot be approved through other existing regulatory pathways.
Why should an animal model developer seek qualification for an animal model?
The animal model qualification process provides a defined framework for the submission, review, and regulatory acceptance of an animal model as a drug development tool (DDT) for use in multiple drug development programs. This framework provides for interactions between FDA and the requestor at key points during the model development process; in particular, interactions during the early stages of animal model development may provide great benefit to the requestor. A qualified animal model is developed independently of any specific drug (i.e., it is product-independent). The use of a qualified model in multiple drug development programs targeting a particular disease or condition reduces redundancy (i.e., multiple drug sponsors developing the same model) and conserves resources (e.g., animals, money, and time).
How will the information about a qualified animal model be made publicly available?
After an animal model is qualified, the information will be made publicly available on the Animal Model Qualification Program website.
Does a requestor retain intellectual property rights for a qualified animal model?
Qualification of an animal model does not trigger any changes to intellectual property rights. After qualification of the animal model, the requestor retains the same intellectual property rights for a qualified animal model as pre-qualification.
What are the benefits to a sponsor using a qualified animal model in drug development?
A qualified animal model may be used in IND or NDA/BLA submissions without FDA needing to reevaluate the qualification conclusion, provided:
- The study is conducted properly (e.g., all procedures and protocols specified in the context of use are followed).
- The animal model is used for the qualified purpose (i.e., used within the context of use).
- At the time of qualification, there is no new information that conflicts with the basis for qualification.
Additionally, the review division will evaluate whether the use of the qualified animal model is suitable for efficacy testing of that particular investigational drug. Integration of qualified animal models into regulatory review can accelerate the drug development and review process.
Is the use of a qualified animal model required for product approval or licensure under the Animal Rule?
No. The use of a qualified model is not required for product approval or licensure under the Animal Rule.
Is a model that was used in the efficacy studies that supported approval of a product under the Animal Rule considered qualified under the Animal Model Qualification Program, based on FDA’s acceptance of its use in product approval?
No. For an animal model to be considered as qualified under the Animal Model Qualification Program, the animal model development studies would still need to be submitted for review and receive a qualification determination.
If a model was used in the efficacy studies that supported approval of a product under the Animal Rule, can that model then be submitted for consideration under the Animal Model Qualification Program?
Yes. Animal model development studies that have already been completed may still be submitted to the Animal Model Qualification Program for review via the Drug Development Tool qualification process.
Do the natural history studies submitted to the program need to be conducted under GLP regulations (21 CFR part 58)?
No. Although FDA recommends the use of GLP regulations, to the extent practicable, for the model-defining natural history studies submitted to support the qualification of an animal model, the use of the GLP regulations is not required, These studies, however, should be conducted in a manner that ensures data quality and integrity, and requestors should provide a description of the data quality and integrity plan that will be used for conducting these studies.
Additional questions can be sent to the AMQP email inbox at CDERAnimalModelQualification@fda.hhs.gov.