Product Development Under FDA's Animal Rule: Understanding FDA's Expectations and Potential Implications for Traditional Development Programs

In 2002 the US Food and Drug Administration (FDA) established a regulatory pathway for drug and biological products targeting indications for which human efficacy studies are not feasible or ethical. These regulations (21 CFR 314.600 for drugs and 21 CFR 601.90 for biologics), commonly referred to as the Animal Rule, were the result of many years of thinking about how to make such products available to people who might need them. A handful of products have been approved under the Animal Rule, and several others are in development. This article reviews how different products met the requirements for licensure under the Animal Rule, based on information publicly available on FDA's website. The primary aim of this manuscript is to offer an understanding of FDA's interpretation of relevant regulations and guidances in the context of this licensure pathway. Some of the methods used for Animal Rule approvals may also have potential application in more traditional development programs. Thus, this article may also offer insight into methods for accelerating product development in general.