Analysis of clinical trials and off-label drug use in hospitalized pediatric patients.

Introduction. The lack of pediatric clinical trials (PCTs) leads to an off-label drug use (OLDU) in children. Our objective was to analyze the number and design of PCTs and OLDU in children in the past years. Population, material and methods. Observational and retrospective study on PCTs and OLDU in children, conducted from 2007 to 2012 in a 252-bed children's hospital. The number and design of PCTs and OLDU in children were analyzed by year and by characteristics. Results. Eighty-seven PCTs and 449 active ingredients corresponding to 1049 drugs prescribed to hospitalized children were evaluated.Of these, 117 (26%) were used off-label. The number of PCTs increased from 2008 to 2011. In 2011, 52.2% of PCTs were non-randomized and uncontrolled studies, and only 39.1% were randomized, controlled trials. Of all studied drugs, 77% corresponded to off-label use. OLDU in children remained steady throughout the study period. Conclusions. In our hospital, the number of pediatric research studies has increased in the past years, being non-randomized and uncontrolled studies the most frequent. OLDU in children has not changed.