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The US Food and Drug Administration (FDA), an agency within the US Department of Health and Human Services (HHS), traces its roots to the 1906 Pure Food and Drug Act. The FDA is responsible for protecting the public health by assuring food (except for meat from livestock, poultry, and some egg products, which are regulated by the US Department of Agriculture) safety and proper labeling; ensuring safety and effectiveness of human and veterinary drugs, vaccines, other biological products, and medical devices; protecting the public from electronic product radiation; ensuring safety and proper labeling of cosmetics and dietary supplements; regulating tobacco products; and finally helping to speed product innovations.1

The federal law establishing the legal framework within which the FDA operates, the Federal Food, Drug, and Cosmetic Act (FDCA), can be found in the US Code (USC) beginning at 21 USC 301. The FDA, similar to other federal agencies, develops regulations based on various laws under which it operates. This process follows the typical procedures required by the Administrative Procedure Act, including “notice and comment rulemaking” allowing public input on the proposed regulation before it is finalized. FDA regulations are also considered federal laws and can be found in Title 21 of the Code of Federal Regulations (CFR). The FDA also issues guidance that is not legally binding on the public or the FDA but describes the agency’s current thinking on regulatory issues.2,3

As evident from the previous description, the FDA is involved in a large portion of the economy. Based on 2019 figures, the FDA is responsible for the oversight of more than $2.6 trillion in consumption, which accounts for about 20 cents of every dollar spent by US consumers.4 Multiple volumes of books have been written on FDA law analyzing every aspect of its function in detail. However, in this chapter, in keeping with this book’s overall theme, the discussion will be limited to the cross section of FDA law that directly affects clinicians. In particular, off-label use, off-label promotion, and preapproval access will be explored in detail. One recurring concept within the scope of this discussion will be that the FDA is not involved in regulating the practice of medicine.

Of note, although within the legal framework of the FDA, drugs and biologics are defined and treated somewhat differently, for the purposes of this chapter, they are used interchangeably. Furthermore, the term medical product will be used in this chapter to describe collectively one or more drug, biologic product, and/or medical device.


Once a drug or device is approved (or cleared) for any specific use, it can be prescribed for any patient and any use as deemed appropriate by the prescriber. Such approach can be used to conduct additional research on that drug or device. It is also commonly done in the regular course of practicing ...

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