Use of Humanized Off-Label Monoclonal Antibodies in Pediatric Practice

Abstract

In recent years, the use of off-label drugs (the use of drugs for indications not approved by state regulatory bodies, and also not mentioned in the instructions for use) has become widespread in the pediatric population. For a long time, clinical trials in the pediatric population were considered difficult to conduct due to ethical issues, leading to frequent off-label drug use. Off-label drug use remains an important public health problem, especially in children with rare diseases or diseases not responding to standard treatment. The purpose of the review is to analyze foreign literature sources on the use of biological preparations outside the instructions. The use of drugs outside the instructions (off-label) is a topical issue in the clinical practice of doctors of various specialties, and this is especially in demand in pediatric practice. This is due to the fact that a registered drug may have a successful experience in adults, but at the same time not have official approval for use in children. The need to use drugs outside the instructions for use, as a rule, occurs in patients with severe, chronic, life-threatening pathology. Further studies analyzing their efficacy, safety, and cost-benefit ratio are needed to expand the use of biological therapy in the pediatric population.