Orphanet J Rare Dis. 2024 Nov 29;19(1):448. doi: 10.1186/s13023-024-03476-4.
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.
PMID:39614329 | DOI:10.1186/s13023-024-03476-4
