Current issues related to the off-label use of drugs and their potential solutions

Relevance. Off-label use of medications is a common practice in clinical settings. To address the growing interest in this issue, the International Society for Pharmacoepidemiology (ISPE) has developed new general guidelines for off-label prescribing in clinical practice that emphasize the importance of evidence-based medicine and promote transparent communication between healthcare professionals and patients. Objective. The aim of this work was to analyze and present key trends for optimizing off-label prescribing decisions based on the ISPE recommendations (2023). Results. The report highlights five main recommendations: seeking strong scientific evidence, including the use of real-world data; using expert knowledge to evaluate and summarize evidence; developing recommendations with rigorous consistency; aligning the use of off-label medications with research; and strengthening collaboration among regulators, researchers, clinicians, and the pharmaceutical industry. Conclusions. A comprehensive approach is required to address the problem of off-label use of medicines. Implementation of these initiatives will reduce the risks associated with off-label use, generate sustainable scientific evidence, and improve the quality of patient care.