Recommendations Developed for Pediatric Multiple Sclerosis Trials
FRIDAY, May 31, 2019 -- The International Pediatric Multiple Sclerosis Study Group has updated standards for future clinical trials in pediatric multiple sclerosis (MS); the new recommendations were published in the May 28 issue of Neurology.
Emmanuelle Waubant, M.D., Ph.D., from the University of California San Francisco MS Center, and colleagues reviewed advances in the understanding of pediatric-onset MS and the advent of clinical trials in this population.
The authors note that convincing evidence has emerged in the last few years that the biological processes involved in MS are mainly shared across the age span. Consequently, there is a biological rationale for using treatments proven efficacious for adults with MS in pediatric MS. There are ethical considerations for conducting clinical trials in this age group, including placebo use in a highly active disease. Regulatory recommendations for future trials in pediatric patients include harmonizing clinical trial design requirements for pediatric therapeutic trials, limiting performance of phase 3 clinical trials to one for a pharmacologic agent, early use of in vitro and animal studies to avoid postponing appropriate testing, and use of pediatric pharmacodynamic/pharmacokinetic studies for all new agents to establish dosage. Future trials in adults should consider enrolling teenagers, and open-label studies should be considered as sufficient for drugs that have been well-studied in adults.
"We are confident these consensus recommendations will help address the need for high-quality evidence to inform the optimal treatment of children and teens living with multiple sclerosis," Waubant said in a statement.
Several authors disclosed financial ties to the pharmaceutical industry.
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Posted: May 2019