Using an approved drug for a disease or medical condition that it was not approved to treat is known as off-label drug use. It is a common occurrence across the nation and has recently increased in popularity with type 2 diabetes medications being used for weight loss. While there are cases of patients benefitting from off-label drug use, there can be repercussions.
Examples of Off-Label Drug Use
The Food and Drug Administration regulates the marketing and safety of drugs and devices but does not regulate how they are prescribed or used. In the United States, off-label drug use accounts for almost one-third of all prescriptions for common drugs and up to 97% in some patient populations.1
Off-label drug use may occur when there is no approved drug to treat the disease or condition, or all other approved treatments may have been tried with no benefit. This may also be the case when there is a lack of development of age-appropriate formulations, i.e., pediatrics. Drugs that are administered differently, such as in a different dosage or by oral solution rather than the approved capsule, are also instances of off-label drug use.2
Glucagon-like peptide-1 (GLP-1s) and sodium-glucose cotransporter 2 (SGLT2) inhibitors are timely examples of off-label drug use. Both were approved for type 2 diabetes use but are often used off-label for weight loss. While the manufacturers of GLP-1s conducted additional studies to approve the drugs’ off-label use for weight loss, SGLT2 inhibitors have not been investigated or approved. Without evidence of their safety and effectiveness, using drugs off-label can come with risks.3
Risks of Off-Label Drug Use
In one study, the off-label use of drugs was associated with a 44% overall higher likelihood of adverse effects in adults. It can lead to unexpected medication interactions causing long-term side effects or conditions to worsen and contribute to drug misuse and abuse. Off-label drug use can also affect drug research efforts by making manufacturers less likely to invest in clinical studies that help assure efficacy and safety in expanded uses.1
In response to the rise of off-label drug use, the International Society for Cell & Gene Therapy (ISCT) established a task force to address practical, ethical, and regulatory issues that arise from the use, and potential misuse, of cell and gene therapies. The Chair of the ISCT Expanded Access Working Group stated, “Inappropriate use of expanded access could harm the entire CGT sector, undermine research efforts, and ultimately delay approval of products critical for many patients. Expanded access is an important mechanism for providing access to unproven, but promising, interventions for seriously and terminally ill patients who lack approved treatment options. However, at the same time, expanded access should not be understood as a mechanism for commercializing unproven, unlicensed interventions.”3
Considerations for Off-Label Drug Use
Healthcare professionals are responsible for prescribing safe and appropriate medications to their patients. However, with the growing use of off-label drugs, there can be inconsistencies in prescribing and safety concerns. If a patient’s healthcare professional is considering an approved drug for an unapproved use, patients should ask the following questions:
- What is the drug approved for?
- Are there other drugs or therapies that are approved to treat my disease or medical condition?
- What scientific studies are available to support the use of this drug to treat my disease or medical condition?
- Is it likely that this drug will work better to treat my disease or medical condition than using an approved treatment?
- What are the potential benefits and risks of treating my disease or medical condition with this drug?
- Will my health insurance cover the treatment of my disease or medical condition with this drug?
- Are there any clinical trials studying the use of this drug for my disease or medical condition that I could enroll in?2
- Van Norman G. A. (2023). Off-Label Use vs Off-Label Marketing of Drugs: Part 1: Off-Label Use-Patient Harms and Prescriber Responsibilities. JACC. Basic to translational science, 8(2), 224–233. https://doi.org/10.1016/j.jacbts.2022.12.011
- Commissioner, O. of the. (n.d.). Understanding unapproved use of approved drugs “Off label.” U.S. Food and Drug Administration. https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/understanding-unapproved-use-approved-drugs-label
- Spencer, D. (2023, July 20). Unlicensed and off-label drugs: What do we know?. Drug Discovery World (DDW). https://www.ddw-online.com/unlicensed-or-off-label-drugs-what-do-we-know-24693-202307/