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Open Access 01-12-2024 | Review

Off-label use of medicines in South Africa: a review

Authors: N. N. Ngcobo, L. J. Mathibe

Published in: Orphanet Journal of Rare Diseases | Issue 1/2024

Abstract

Background

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.

Aim

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.

Conclusion

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.

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Title: Off-label use of medicines in South Africa: a review
Authors: N. N. Ngcobo
L. J. Mathibe
Publication date: 01-12-2024
Publisher: BioMed Central
Published in: Orphanet Journal of Rare Diseases / Issue 1/2024
Electronic ISSN: 1750-1172
DOI: https://doi.org/10.1186/s13023-024-03476-4

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TARS2 c.470 C > G is a chinese-specific founder mutation in three unrelated families with mitochondrial encephalomyopathy

Comprehensive insights into health services accessibility and quality of life of families with individuals with 22q11.2 deletion syndrome in Brazil

Assessing the diagnostic utility of the Gaucher Earlier Diagnosis Consensus (GED-C) scoring system using real-world data

Structural brain abnormalities in Pallister-Killian syndrome: a neuroimaging study of 31 children

Long-term safety and influence on growth in patients receiving sirolimus: a pooled analysis

A review of multiple diagnostic approaches in the undiagnosed diseases network to identify inherited metabolic diseases

Keynote series | Spotlight on menopause

Springer Medicine

Abstract

Background

Aim

Conclusion

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TARS2 c.470 C > G is a chinese-specific founder mutation in three unrelated families with mitochondrial encephalomyopathy

Comprehensive insights into health services accessibility and quality of life of families with individuals with 22q11.2 deletion syndrome in Brazil

Assessing the diagnostic utility of the Gaucher Earlier Diagnosis Consensus (GED-C) scoring system using real-world data

Structural brain abnormalities in Pallister-Killian syndrome: a neuroimaging study of 31 children

Long-term safety and influence on growth in patients receiving sirolimus: a pooled analysis

A review of multiple diagnostic approaches in the undiagnosed diseases network to identify inherited metabolic diseases