title: Children with multiple sclerosis should not become therapeutic hostages.
abstract: BACKGROUND
  Both the United States (US) Food and Drug Administration (FDA) and the European Union (EU) European Medicines Agency (EMA) order pediatric clinical trials as a condition for approval of new compounds. We evaluate clinical value and likelihood of sufficient recruitment for pediatric multiple sclerosis (pMS) studies and discuss US and EU pediatric legislation with pMS as a paradigm.
  
  
  METHODS
  We analyzed pMS clinical trials requested by the FDA and the EMA and industry-sponsored pMS studies registered on www.clinicaltrials.gov and www.clinicaltrialsregister.eu.
  
  
  RESULTS
  The FDA demands four and the EMA 15 pMS trials.
  
  
  CONCLUSIONS
  pMS is rare. Neither FDA nor EMA prioritize compounds for potential benefit in pMS. The EMA in particular orders multiple pMS studies, which will probably not recruit enough patients. Therefore, it is likely that the pMS trial outcomes will not be relevant for evidence-based medicine analyses, clinical practice and a pMS label for the respective drug. EMA requests for multiple pediatric studies have been described in metastasized adolescent melanoma, another very rare pediatric disease. The terms 'ghost studies' and 'therapeutic hostages' have been proposed for such trials and children whose parents are lured into permitting study participation. Clinical studies are not ethical if the probability is high that they will not provide reasonable outcomes. For now, pMS clinicians will have to continue to use new MS drugs in children off-label. They might consider a more proactive international coordinating role in prioritizing and testing new MS compounds in children.
authors: Klausrose Consulting, Pediatric Drug Development and More, Aeussere Baselstrasse 308, 4125 Riehen, Switzerland.
authors: Klaus
authors: K
authors: Rose
authors: Department of Neurology, St Joseph Hospital Berlin-Weissensee, Berlin, Germany.
authors: Thomas
authors: T
authors: Müller
keywords: better medicines for children; multiple sclerosis; pediatric clinical trials; pediatric drug development; pediatric legislation