Disease-modifying drugs (DMDs) for multiple sclerosis (MS) have been evaluated in pediatric patients in observational studies demonstrating a similar, even better clinical effect compared to adults, with a similar safety. Only fingolimod has been tested in a randomized controlled trial (RCT) and is approved for pediatric multiple sclerosis (ped-MS). Numerous methodological, practical, and ethical issues underline that RCTs are difficult to conduct in ped-MS. This also creates a lack of safety information. To facilitate the availability of new agents in ped-MS, we encourage to develop a different approach based on pharmacokinetic/pharmacodynamic studies to yield information on optimal doses and implementation of obligatory registries to obtain information on safety as primary endpoint.

Centro Studi Sclerosi Multipla Ospedale di Gallarate Gallarate Italy

CIC 1414 INSERM Department of Neurology CHU Rennes Rennes France

Department of Basic Medical Sciences Neurosciences and Sense Organs University of Bari Bari Italy

Department of NEUROFARBA University of Florence Florence Italy; IRCCS Fondazione Don Carlo Gnocchi Florence Italy

Department of Neurology and Center for Clinical Neuroscience 1st Medical Faculty Charles University Prague Czech Republic

Department of Neurology Danish Multiple Sclerosis Center Copenhagen University and Rigshospitalet Copenhagen Denmark

Department of Neurology UKD and Center of Neurology and Neuropsychiatry Heinrich Heine University Düsseldorf Germany

Istituto di Neurologia Sperimentale IRCCS Ospedale San Raffaele Milan Italy

Multiple Sclerosis Center Sant' Andrea Hospital Rome Italy

Neurologic Clinic and Policlinic Departments of Medicine Clinical Research Biomedicine and Biomedical Engineering University Hospital and University of Basel Basel Switzerland

St Michael's Hospital University of Toronto Toronto ON Canada; Department of Neurology Cemcat Hospital Vall d'Hebron Barcelona Spain

University of Lille INSERM UMR S1172 CHU Lille FHU Imminent Lille France

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