Medicamentos off-label e não licenciados no âmbito pediátrico: uma revisão descritiva dos últimos dez anos

Abstract

The lack of drugs containing adequate dosages for the treatment of pediatric patients is a problem frequently observed in hospitals, especially in the pediatric and neonatal intensive care unit (ICU) sectors. It is known that, for a treatment to be considered safe and effective, series of clinical studies are necessary, however, a low number of these studies involving children are reported, mainly due to ethical issues that make their conduct difficult. Thus, few drugs prove to be suitable for the treatment of these patients, making it necessary to resort to the use of off-label and unlicensed drugs. Drugs are classified as off-label when their use differs from their approved specifications, in turn, unlicensed products are classified in this way because they do not have approval for marketing in the country or do not have proof of safety and efficacy. Preparation of organized study protocols, reporting information to parents and the child in a clear and objective way, bringing researchers and parents closer to establish a relationship of trust and conducting research at moments when the family is available are important strategies to facilitate the conduct of clinical trials.

Keywords

• off-label
• não licenciado
• pediatria
• estudos clínicos pediátricos