ABSTRACT
This chapter examines the ethical issues surrounding off-label use. The issues include the balance between Food and Drug Administration (FDA) roles in protecting public safety and promoting innovation, FDA’s role in regulating the promotion of off-label uses, ethical and liability issues stemming from off-label use in the clinical context, and questions regarding access to experimental therapies. FDA’s regulatory jurisdiction does not extend to the actual practice of medicine. The practice of medicine is regulated by state licensing laws, which grant physicians the autonomy to practice medicine in accordance with their own professional judgment. The off-label use exception recognizes that innovation runs far apace of the FDA approval process; it allows research and science to flourish within the context of a cumbersome regulatory scheme. The trade-off of the off-label use exception is that it undermines FDA’s ability to ensure either the safety or efficacy of nonapproved uses and it reduces the information about the product’s safety and efficacy.
