Medicamentos novos registrados no Brasil de 2003 a 2013: análise na perspectiva da atenção à criança

Abstract

Medicines is an important health technology in different levels of child health care. However, medications in certain pediatric clinical conditions are used off-label or unlicensed, due to the unavailability of appropriate medicines. The aim of this research was to analyze the new drugs registered in Brazil, in the period from January 1st, 2003 to december 31st, 2013, from the perspective of child care, considering burden of disease, suitability to the target age range and safety. Retrospective cohort study with documental analysis in databases, technical and scientific publications and official documents of drug regulatory agencies, as well as the Health Ministry and the World Health Organization. Drugs characteristics, pediatric indication at the time of registration abroad, the inclusion of pediatric indication and the expansion of the age bracket for children, after registration, were investigated. In order to evaluate therapeutic innovation level of the new drugs, the algorithm of Motola et al. 2004 was used. The indications for the drugs were related to the Disability-Adjusted Life Year (DALY) from the 2015 Global Burden of Disease Study. The evaluation of the drug suitability to the pediatric age group was carried out using the following criteria: i. drug dosage form suitability; ii. ability to provide the recommended dose. Drugs that met the two criteria were considered appropriate for the pediatric age groups. Postmarketing safety alerts for the pediatric population have been identified in the WHO Drug Information Bulletin and on the website of drug regulatory agencies. A descriptive statistical analysis was performed. The correlation between the number of new drugs and DALY, of children younger than 15 years old, was determined by the Spearman´s coefficient. In the cohort, 134 new drugs were included and 46 (34.3%) had a pediatric indication. None of these medicines had a specific indication for children. Only 9 (19.6%) of the drugs presented important therapeutic innovation. The number of new drugs with a pediatric indication at the time of registration was small, reflecting the international challenges in developing effective and safe medicines for children. There is no relation between the number of new drugs and burden of disease. Adequacy due to the drug's ability to provide the recommended dose was greater than 80% across all age groups. Regarding the suitability of the drug dosage form, it was identified that the higher the age group, the greater the proportion of adequacy. The high prevalence of solid dosage forms contributed to the inadequacy of the medicines to the pediatric age group. The publication of postmarketing safety alerts needs to be expanded in order to reduce the risks of adverse reactions and increase knowledge about drug safety.