Off-label Drug use literature review article

Nonn, Ahmed Mansour; Mohamed, Esraa; Al-ansary, Sohair Lotfy

doi:10.21608/jpsdm.2025.296241.1018

Off-label Drug use literature review article

Document Type : Review Articles.

Authors

¹ Department of Pharmaceutical Chemistry, College of Pharmaceutical Sciences and Drug Manufacturing , Misr university for Science and Technology , October 6 city , Egypt

² Department of Pharmacognosy, College of Pharmaceutical Sciences and Drug Manufacturing, Misr University for Science and Technology

10.21608/jpsdm.2025.296241.1018

Abstract

Off-label drug use (OLDU) refers to prescribing medications for conditions not approved by regulatory bodies like the FDA. Though widely used in medical practice, many healthcare professionals remain underinformed about OLDU, which is common in cases where conventional therapies fail, such as resistant, idiopathic, or orphan diseases. Currently, OLDU represents around 21% of prescriptions, with projections suggesting it could reach 30% in the U.S. by 2030. Key categories of drugs used off-label include anticancer, cardiac, anticonvulsant, anti-asthmatic, allergy, psychiatric, peptic ulcer, antimicrobial, antihypertensive, and women’s health therapies. The practice is most prevalent in Western countries, affecting pediatric, oncology, critical care, and pregnancy patients the most.

However, OLDU poses various challenges, including limited clinical evidence, regulatory and legal uncertainties, patient safety risks, insurance complications, ethical concerns, and inadequate post-market surveillance. Addressing these issues is crucial to ensuring OLDU's safe and effective application. Once a drug is used off-label, the manufacturer must conduct further clinical trials and stability tests to potentially secure approval for the new indication. Without FDA approval, challenges include limited reimbursement by insurers, the potential for unforeseen adverse effects, and uncertainties about therapeutic effectiveness.

Despite these risks, OLDU has played a significant role in managing complex cases and addressing urgent needs, such as during the COVID-19 pandemic and in cancer treatments. Ultimately, the decision to use off-label drugs depends on patient history and clinical context, with careful consideration by healthcare providers. OLDU continues to provide critical therapeutic options up to 2024.

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