title: International Pediatric MS Study Group Clinical Trials Summit: meeting report.
abstract: OBJECTIVE
  Pediatric studies for new biological agents are mandated by recent legislation, necessitating careful thought to evaluation of emerging multiple sclerosis (MS) therapies in children with MS. Challenges include a small patient population, the lack of prior randomized clinical trials, and ethical concerns. The goal of this meeting was to assess areas of consensus regarding clinical trial design and outcome measures among academic experts involved in pediatric MS care and research.
  
  
  METHODS
  The Steering Committee of the International Pediatric MS Study Group identified key focus areas for discussion. A total of 69 meeting attendees were assembled, including 35 academic experts. Regulatory and pharmaceutical representatives also attended, and provided input, which informed academic expert consensus decisions.
  
  
  RESULTS
  The academic experts agreed that clinical trials were necessary in pediatric MS to obtain pharmacokinetic, safety and efficacy data, and regulatory approval allowing for greater medication access. The academic experts agreed that relapse was an appropriate primary outcome measure for phase III pediatric trials. An international standardized cognitive battery was identified. The pros and cons of various trial designs were discussed. Guidelines surrounding MRI studies, pharmacokinetics, pharmacodynamics, and registries were developed. The academic experts agreed that given the limited subject pool, a stepwise approach to the launch of clinical trials for the most promising medications is necessary in order to ensure study completion. Alternative approaches could result in unethical exposure of patients to trial conditions without gaining knowledge.
  
  
  CONCLUSIONS
  Consensus points for conduct of clinical trials in the rare disease pediatric MS were identified amongst a panel of academic experts, informed by regulatory and industry stakeholders.
authors.affiliation: Partners Pediatric Multiple Sclerosis Center, Massachusetts General Hospital, Boston, MA, USA. tchitnis@partners.org
authors.forename: Tanuja
authors.initials: T
authors.lastname: Chitnis
authors.forename: Marc
authors.initials: M
authors.lastname: Tardieu
authors.forename: Maria Pia
authors.initials: MP
authors.lastname: Amato
authors.forename: Brenda
authors.initials: B
authors.lastname: Banwell
authors.forename: Amit
authors.initials: A
authors.lastname: Bar-Or
authors.forename: Angelo
authors.initials: A
authors.lastname: Ghezzi
authors.forename: Andrew
authors.initials: A
authors.lastname: Kornberg
authors.forename: Lauren B
authors.initials: LB
authors.lastname: Krupp
authors.forename: Daniela
authors.initials: D
authors.lastname: Pohl
authors.forename: Kevin
authors.initials: K
authors.lastname: Rostasy
authors.forename: Silvia
authors.initials: S
authors.lastname: Tenembaum
authors.forename: Emmanuelle
authors.initials: E
authors.lastname: Waubant
authors.forename: Evangeline
authors.initials: E
authors.lastname: Wassmer
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods / Q000592:standards*; D003245:Consensus Statements as Topic; D004219:District of Columbia; D006801:Humans; D007155:Immunologic Factors / Q000627:therapeutic use; D038622:Internationality; D009103:Multiple Sclerosis / Q000453:epidemiology / Q000628:therapy*; D010372:Pediatrics / Q000379:methods / Q000592:standards*; D058028:Research Report / Q000592:standards*
chemical_list: D007155:Immunologic Factors