Discipline of Pharmaceutical Sciences, School of Health Sciences, University of KwaZulu-Natal, Durban, South Africa.
Division of Pharmacology (Therapeutics), Discipline of Pharmaceutical Sciences, Nelson R. Mandela School of Medicine, University of KwaZulu-Natal, Durban, South Africa.
Orphanet J Rare Dis. 2024 Nov 29;19(1):448. doi: 10.1186/s13023-024-03476-4.
Off-label use of medicinal products has become an important part of mainstream and legitimate medical practice worldwide. This practice is common in oncology, obstetrics, paediatrics, and in the management of infectious diseases (notably HIV), and inflammatory conditions as well as in rare and/or orphan diseases. However, the off-label use of medicines recently-raised many clinical and legal difficulties, not only among medical practitioners but also among pharmacists and other healthcare professionals.
This paper, therefore, highlights the advantages (such as cost saving for both the patient and the country/insurance that is paying for the medication) and disadvantages (insufficient evidence available) of the use of medicines to treat specific conditions or indications for which they are currently not registered.
Off-label drug use can be likened to a double-edged sword, offering valuable opportunities for medical practitioners while carrying potential risks. When the scientific basis for off-label use is unclear, it may place patients at risk of unapproved experimentation, unforeseen health hazards, and ineffective treatments. Hence, there is a pressing need in South Africa for clear regulatory guidelines on off-label drug use. Additionally, the timely review and approval of new indications for medicines, backed by robust scientific evidence, are essential. This would reduce the significant burden and inherent risks faced by medical practitioners when using medicines off-label to provide compassionate care.
药品的标签外使用已成为全球主流且合法医疗实践的重要组成部分。这种做法在肿瘤学、妇产科、儿科以及传染病（尤其是 HIV）和炎症性疾病的管理中很常见，也存在于罕见病和/或孤儿病中。然而，药品的标签外使用最近引发了许多临床和法律上的困难，不仅在医疗从业者中，在药剂师和其他医疗保健专业人员中也是如此。
因此，本文强调了使用药品治疗特定病症或适应症（这些药品目前尚未注册）的优势（例如为患者和支付药物费用的国家/保险公司节省成本）和劣势（证据不足）。
药品的标签外使用可以比作一把双刃剑，为医疗从业者提供了宝贵的机会，但也存在潜在的风险。当标签外使用的科学依据不明确时，患者可能面临未经批准的实验、意料之外的健康危害和无效治疗的风险。因此，南非迫切需要制定关于药品标签外使用的明确监管指南。此外，及时审查和批准有充分科学证据支持的新药品适应症至关重要。这将减轻医疗从业者在使用药品标签外时提供同情护理所面临的重大负担和固有风险。
