Public disclosure of clinical trials in children

Background: Although randomized controlled trials (RCTs) are considered the most reliable way to assess a drug's safety and effectiveness, they are not always necessary or feasible for assessing the effects of health care, on pediatric patients. Recent legislation passed in the United States has led to an increase in the number of RCTs in pediatric populations, but the drugs studied in these trials have not always proven useful to pediatric patients. Objective: This study was undertaken to identify the specific areas addressed by clinical research in the European pediatric therapeutic field. Methods: A literature search was conducted on EMBASE for therapeutic clinical trials in children, performed in the 15 European Union countries and published from 1995 to 2000, using the search terms child, infant, clinical trial, comparative studies, drug, and drug therapy. A general analysis of all the studies was performed, as well as a more in-depth analysis of the RCTs. Two researchers reviewed each study independently using a standard form. Data on methodology, diseases, and outcomes were extracted. All studies published in 5 general pediatric journals were assessed for reporting status of ethical committee approval and informed consent. Results: A total of 338 articles were identified as being therapeutic, pediatric, clinical trials. Of these, 139 were RCTs. These 139 studies were performed in 12 countries; most addressed the treatment of asthma, acute otitis media, acute upper and lower respiratory infections, and vaccine-preventable diseases. Of the 37 studies published in 5 chosen general pediatrics journals, 24 and 30 did not report ethical committee approval or informed consent, respectively. Conclusions: The diseases addressed by the RCTs identified in this review are among the leading causes of burden of disease in European children. However, ample knowledge of the physiopathologic and therapeutic variables of these diseases already exists. More research is needed on new drugs and disease states as they relate to pediatric patients. Moreover, information about these trials must be made readily available,to parents and family physicians, both to increase enrollment and to uphold the highest ethical standards of providing optimal treatment to all patients.