Wednesday, 27 March 2013

Childhood MS

#MSBlog: Childhood MS in the spotlight.

"This report is timely can confirms the MS communities commitment to children with MS and the need to get DMTs approved for treating childhood MS. Hopefully, parents of children with MS will find this initiative a move in the right direction."

Chitnis et al. International Pediatric MS Study Group Clinical Trials Summit: Meeting report. Neurology. 2013 Mar;80(12):1161-1168.


OBJECTIVE: Paediatric 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.

CONCLUSION: Consensus points for conduct of clinical trials in the rare disease paediatric MS were identified amongst a panel of academic experts, informed by regulatory and industry stakeholders.



1 comment:

  1. Data for pediatric MS treatment is certainly needed so trials have to be done.

    But many things are still unclear. Currently children get the same treatments as adults do. In the future will treatment be delayed till pediatric trials are done?
    Will pediatric trials be held for all the current treatments?

    Ref 'The academic experts agreed that relapse was an appropriate primary outcome measure for phase III pediatric trials':
    My daughter went two years with no relapses but with every MRI showing lots of new lesions.

    ReplyDelete