Clinical trials in pediatric multiple sclerosis: overcoming the challenges

To date, none of the formative clinical drug trials in multiple sclerosis (MS) have included children. Just as in other fields in pediatrics, current prescribed therapies are off-label and are based on the results of adult studies. Emerging oral, injectable and intravenous immunotherapies appear to be more efficacious, but simultaneously have more worrisome side effects. In order to optimize therapy for children with MS, these therapies must be evaluated in robust clinical trials with a focus on monitoring for potential toxicities both in the short and long term. Many challenges exist in conducting clinical trials in children, including smaller patient populations. International collaboration and mandated pediatric investigation plans will facilitate the process of conducting these valuable clinical trials, which may lend to a better understanding of MS overall.