Initiation and management of a pediatric multicenter drug trial by an academic center

Abstract

Background: Although the number of prospective pediatric drug trials has increased in the past few years, there are still fewer studies done in pediatric patients than in adults. Such studies are critical for determining the safety, efficacy, and appropriate dosing of drugs in children. Objectives: This article outlines the difficulties encountered in initiating and performing multicenter drug studies in children and offers recommendations for developing and conducting such studies. Methods: We reviewed existing literature in pediatric drug trials, searching PubMed and reviewing topics in such trials, and analyzing our own experience. Results: Recent legislation offers financial incentives to pharmaceutical companies to conduct clinical trials in pediatric patients; however, obstacles to the successful initiation and completion of such trials still exist. Pediatric centers are often inexperienced at developing clinical trial protocols and applying for research grants. In addition, many drugs cannot be administered at appropriate doses to smaller patients in tablet or capsule form, making it necessary to develop new formulations. Furthermore, parents may be reluctant to allow their children to participate in placebo-controlled trials. Pediatricians interested in developing drug trials must plan carefully to anticipate all of the budgetary, staffing, regulatory, and enrollment problems that may arise over the course of such a trial. Conclusions: More trials are needed to determine the safety, efficacy, and optimal doses of new drugs and of drugs already used off-label to treat pediatric patients. The financial incentives offered by legislation provide additional reasons for pediatricians and industry to work together to perform drug trials in pediatric patients.