Scoping review of the availability and uptake of disease modifying therapies in children and adolescents with multiple sclerosis.

Abstract

Introduction: Approximately 10% of individuals with multiple sclerosis (MS) have pediatric-onset (<18-years-old). Pediatric-specific barriers to accessing disease modifying therapies (DMT) exist. Issues include few pediatric-based randomized controlled trials (RCT), often required for formal regulatory approval, and resultant challenges with cost/coverage. This review assessed real-world DMT uptake in pediatric-MS to better understand potential barriers.

Areas covered: We performed a scoping review of observational studies examining DMTs in patients with pediatric-MS published between 07/1993-06/2024. PRISMA guidelines were used. Databases searched included: Cochrane Library, Ovid MEDLINE/Embase, Scopus, and Web of Science. Studies must include > 10 DMT exposed pediatric-MS patients with full-text available in English. RCTs/pharmaceutical-industry funded studies were excluded. Of 2114 abstracts screened, 88 studies were included. A total of 21,591 patients (13,411 females) were included. DMTs were used in 68.7% (n = 14,833). Most studies were from Europe (53.4%), North America (22.7%), or the Middle East (10%). Regional variabilities were found in DMT uptake between continents. Only 13 (14.8%) studies included information on DMT funding source.

Expert opinion: Pediatric-MS patients showed low DMT uptake with variability in DMT use based on region. Limited data was found regarding specific barriers to DMT access. Further research is needed to better understand regional barriers to access.