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Off-Label Medication: From a Simple Concept to Complex Practical Aspects

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Rusz, Carmen-Maria, Ősz, Bianca-Eugenia, Jîtcă, George, Miklos, Amalia, Bătrînu, Mădălina-Georgiana, Imre, Silvia·2021·International Journal of Environmental Research and Public Health
DOI 10.3390/ijerph181910447PMC PMC8508135
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Abstract

Off-label use of drugs is widely known as unapproved use of approved drugs, and it can be perceived as a relatively simple concept. Even though it has been in existence for many years, prescribing and dispensing of drugs in an off-label regimen is still a current issue, triggered especially by unmet clinical needs. Several therapeutic areas require off-label approaches; therefore, this practice is challenging for prescribing physicians. Meanwhile, the regulatory agencies are making efforts in order to ensure a safe practice. The present paper defines the off-label concept, and it describes its regulation, together with several complex aspects associated with clinical practices regarding rare diseases, oncology, pediatrics, psychiatry therapeutic areas, and the safety issues that arise. A systematic research of the literature was performed, using terms, such as “off-label”, ”prevalence”, ”rare diseases”, ”oncology”, ”psychiatry”, ”pediatrics”, and ”drug repurposing”. There are several reasons for which off-label practice remains indispensable in the present; therefore, efforts are made worldwide, by the regulatory agencies and governmental bodies, to raise awareness and to ensure safe practice, while also encouraging further research.

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  • Studies confirm the prevalence of off-label use in rare diseases, such as oncology and psychiatry, particularly among children and the elderly.

    “This aligns with the European Committee’s report on off-label use, as studies confirm its prevalence in rare diseases, such as oncology and psychiatry, particularly among children and the elderly.159”

    From Off-label and repurposed use of disulfiram beyond alcohol dependence: A systematic review of clinical and preclinical evidence (2026) · cites this paper as [159]

    The Introduction reports that off-label use is widespread in rare diseases, oncology, pediatrics, and psychiatry, especially among pediatric and elderly psychiatric patients. The claim conflates these separate categories by presenting oncology and psychiatry as examples of rare diseases; the Rare Diseases section identifies only specific cancers as rare diseases. (LLM, conf=0.82)

    Experimental — accuracy not yet measured

  • The key benefit of off-label prescribing is addressing unmet medical needs where standard treatments fall short, thereby broadening medication access for vulnerable patient groups.

    “The key benefit of off-label prescribing is addressing unmet medical needs where standard treatments fall short, thereby broadening medication access for vulnerable patient groups.159”

    From Off-label and repurposed use of disulfiram beyond alcohol dependence: A systematic review of clinical and preclinical evidence (2026) · cites this paper as [159]

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Authors
Rusz, C.-M., Ősz, B.-E., Jîtcă, G., Miklos, A., Bătrînu, M.-G., & Imre, S.
Affiliations
Universitatea de Medicină, Farmacie, Științe și Tehnologie „George Emil Palade” din Târgu Mureș (RO)
Corresponding author
Ősz, Bianca-Eugenia
ORCID
Venue
International Journal of Environmental Research and Public Health
Publisher
MDPI AG
Published
2021-10-04
Volume
18
Issue
19
Pages
10447
ISSN
1660-4601 (ISSN-L)
DOI
10.3390/ijerph181910447
PMC
PMC8508135
OpenAlex
W3204211831
Cited by
58
References
87
Citations by year
2026: 7 · 2025: 14 · 2024: 14 · 2023: 14 · 2022: 9
FWCI
2.24
Type
journal article
Language
en
Open access
Licence
Locations
  • International Journal of Environmental Research and Public Health (publishedVersion, cc-by)
  • PubMed Central (publishedVersion, cc-by)
  • PubMed (publishedVersion)
  • RePEc: Research Papers in Economics (submittedVersion)
  • DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion)
Topics
  • Health Sciences › Medicine › Pediatrics, Perinatology and Child Health › Pharmaceutical studies and practices
  • Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Pharmaceutical Economics and Policy
  • Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmacology › Pharmaceutical industry and healthcare
Keywords
off-label drug use, drug repurposing, rare diseases, drug safety, unmet clinical needs, prescribing patterns
MeSH terms
Child, Humans, Medical Oncology, Physicians, Psychiatry, Prevalence, 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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