Portal de Periódicos da CAPES

Peer-Reviewed Publication of Clinical Trials Completed for Pediatric Exclusivity

2006; American Medical Association; Volume: 296; Issue: 10 Linguagem: Inglês

10.1001/jama.296.10.1266

1538-3598

Daniel K. Benjamin, Philip Brian Smith, M. Dianne Murphy, Rosemary Roberts, Lisa L. Mathis, Debbie Avant, Robert M. Califf, Jennifer S. Li,

Health Systems, Economic Evaluations, Quality of Life

ContextMuch of pediatric drug use is off-label because appropriate pediatric studies have not been conducted and the drugs have not been labeled by the US Food and Drug Administration (FDA) for use in children. In 1997, Congress authorized the FDA to grant extensions of marketing rights known as "pediatric exclusivity" if FDA-requested pediatric trials were conducted. As a result, there have been over 100 product labeling changes. The publication status of studies completed for pediatric exclusivity has not been evaluated.ObjectiveTo quantify the dissemination of results of studies conducted for pediatric exclusivity into the peer-review literature.DesignCohort study of all trials conducted for pediatric exclusivity between 1998 and 2004 as determined by MEDLINE and EMBASE searches through 2005, the subsequent labeling changes, and the publication of those studies in peer-reviewed journals. We categorized any labeling changes resulting from the studies as positive or negative for the drug under study. We then evaluated aspects of the studies and product label changes that were associated with subsequent publication in peer-reviewed medical journals.Main Outcome MeasuresPublication of the trial data in peer-reviewed journals.ResultsBetween 1998 and 2004, 253 studies were submitted to the FDA for pediatric exclusivity: 125 (50%) evaluated efficacy, 51 (20%) were multi-dose pharmacokinetic, 34 (13%) were single-dose pharmacokinetic, and 43 (17%) were safety studies. Labeling changes were positive for 127/253 (50%) of studies; only 113/253 (45%) were published. Efficacy studies and those with a positive labeling change were more likely to be published.ConclusionsThe pediatric exclusivity program has been successful in encouraging drug studies in children. However, the dissemination of these results in the peer-reviewed literature is limited. Mechanisms to more widely disperse this information through publication warrant further evaluation.