Clinical trials of drugs in the pediatric population

This paper reviews current trends in pediatric clinical trials conducted for drugs in the United States. There are many known challenges in conducting clinical trials in the pediatric population, including the lack of available patients and financial incentives, among others. In this paper, we found that the majority of marketed drugs are also approved in the United States for pediatric patients, which indicates that various government incentives and/or requirements are effective in encouraging pharmaceutical companies to conduct pediatric clinical trials. However, a gap still exists for some pediatric diseases. Based on our review, the rarity of diseases in the pediatric population is one of the main factors in determining whether pediatric clinical trials are conducted for a given medication. For these diseases, further government incentives and requirements are unlikely to be effective in promoting pediatric clinical trials because of the inherent difficulties associated with the limited number of patients and the severity of the disease. While not ideal, we propose that adopting alternative clinical protocols suited for a limited cohort as well as using relevant biomarkers and the quality of life (QOL) as primary clinical endpoints may encourage and expedite clinical trials in pediatric patients. FDA guidance on efficacy age-agnostic scientific and technological platforms may incentivize expanding the use of approved drugs in pediatric patients with only pharmacokinetic supporting data. In addition, instituting a requirement to report off-label (pediatric) use to a centralized database may provide valuable information for physicians to use relevant medications for treating pediatric patients with such diseases.