Ten common questions (and their answers) about off-label drug use
Open accessAbstract
The term off-label drug use (OLDU) is used extensively in the medical literature, continuing medical education exercises, and the media. Yet, we propose that many health care professionals have an underappreciation of its definition, prevalence, and implications. This article introduces and answers 10 questions regarding OLDU in an effort to clarify the practice's meaning, breadth of application, acceptance, and liabilities. Off-label drug use involves prescribing medications for indications, or using a dosage or dosage form, that have not been approved by the US Food and Drug Administration. Since the Food and Drug Administration does not regulate the practice of medicine, OLDU has become common. It occurs in every specialty of medicine, but it may be more common in areas of medicine in which the patient population is less likely to be included in clinical trials (eg, pediatric, pregnant, or psychiatric patients). Pharmaceutical companies are not allowed to promote their medications for an off-label use, which has lead to several large settlements for illegal marketing. To limit liability, physicians should prescribe medications only for indications that they believe are in the best interest of the patient. In addition, health care professionals should educate themselves about OLDU to weigh the risks and benefits and provide the best possible care for their patients.
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Physicians may treat off-label clinical conditions and diseases for patients that respond poorly to conventional treatment, often in specific populations poorly represented in clinical trials, e.g. pediatric, pregnant or psychiatric patients.
“physicians may treat off-label clinical conditions and diseases for patients that respond poorly to conventional treatment, often in specific populations poorly represented in clinical trials, e.g. pediatric, pregnant or psychiatric patients (16).”
From DrugCentral 2023 extends human clinical data and integrates veterinary drugs (2023) · cites this paper as [16]
Using pharmaceutical formulations or dosage not mentioned in the drug label for an indication falls into the practice of off-label drug use.
“Using pharmaceutical formulations (e.g. oral solution instead of capsules) or dosage (e.g. two tablets instead of one per day) not mentioned in the drug label for an indication also falls into the practice of off-label drug use (16).”
From DrugCentral 2023 extends human clinical data and integrates veterinary drugs (2023) · cites this paper as [16]
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- Authors
- Wittich, C. M., Burkle, C. M., & Lanier, W. L.
- Affiliations
- Mayo Clinic (US)
- Corresponding author
- Wittich, Christopher M.
- Venue
- Mayo Clinic Proceedings
- Publisher
- Elsevier BV
- Published
- 2012-10
- Volume
- 87
- Issue
- 10
- Pages
- 982-990
- ISSN
- 0025-6196 (ISSN-L)
- PMC
- PMC3538391
- OpenAlex
- W2166119767
- Cited by
- 368
- References
- 48
- Citations by year
- 2026: 17 · 2025: 24 · 2024: 62 · 2023: 42 · 2022: 32 · 2021: 35 · 2020: 26 · 2019: 29 · 2018: 25 · 2017: 25 · 2016: 21 · 2015: 11 · 2014: 12 · 2013: 4 · 2012: 3
- FWCI
- 8.41
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Mayo Clinic Proceedings (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Pediatrics, Perinatology and Child Health › Pharmaceutical studies and practices
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmacology › Pharmaceutical industry and healthcare
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Pharmaceutical Economics and Policy
- Keywords
- off-label drug use, FDA-approved indications, prescribing patterns, physician liability, pharmaceutical marketing, pediatric prescribing, pregnant patients, psychiatric patients, risk-benefit assessment
- MeSH terms
- Attitude of Health Personnel, Drug Labeling, Female, Humans, Male, Patient Education as Topic, Physician-Patient Relations, Practice Patterns, Physicians', Drug Prescriptions, United States, United States Food and Drug Administration, Safety Management, Off-Label Use
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Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
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