Globalization of pediatric research: analysis of clinical trials completed for pediatric exclusivity

We evaluated the setting of published studies conducted under the US Pediatric Exclusivity Provision, which provides economic incentives to pharmaceutical companies to conduct drug studies with children.

Published studies containing the main results of trials conducted in 1998-2007 under the Pediatric Exclusivity Provision were included. Data were extracted from each study and described, including the therapeutic area of drug studied, number of patients enrolled, number of sites, and location where the study was conducted, if reported.

Overall, 174 trials were included (sample size: 8-27 065 patients); 9% did not report any information regarding the location or number of sites where the study was conducted. Of trials that did report such information, 65% were conducted in >or=1 country outside the United States, and 11% did not include any sites in the United States. Fifty-four countries were represented, and 38% of trials enrolled patients in >or=1 site located in a developing/transition country, including more than one-third of infectious disease, cardiovascular, and allergy/immunology trials.

The majority of published pediatric trials conducted under the Pediatric Exclusivity Provision included sites outside the United States, and more than one-third of trials enrolled patients in developing/transition countries.