Appropriate off-label drug use

ABSTRACT

After new drugs are introduced into an area of medical practice through the regulatory approval process, clinicians gain further experience using the drugs and amass real-world evidence regarding their efficacy. For these reasons, drugs can be prescribed in contexts extending beyond their labeled indication, dosage, or route because of unmet needs or additional benefits, such as cost-effectiveness. Off-label drug use (OLDU) is widespread in various clinical fields, especially in pediatrics, obstetrics, psychiatry, and oncology; however, the small amount of supporting evidence associated with increased adverse drug events has emerged as an important issue regarding OLDU. Nonetheless, OLDU has officially been allowed in South Korea by an announcement of the Ministry of Health and Welfare, a public announcement of the Health Insurance Review and Assessment Service for cancer drugs, and through submissions to the Health Insurance Review and Assessment Service by hospitals after receiving institutional review board approval. Despite these systems, the proper scope of OLDU has been a topic of debate. In order to improve the appropriateness of OLDU, a transparent system for evidence-based recommendations, conditional decision-making, and monitoring of adverse drug events should be established and organized in collaboration with existing systems to promote consistent decision-making.