Abstract
The population with multiple sclerosis receiving treatment in clinical practice differs from that in randomized controlled trials (RCTs). An assessment of the real-world benefit–risk profile of therapies is needed. This analysis used data from the large, noninterventional, observational German study Post-Authorization Non-interventional German sAfety study of GilEnyA (PANGAEA) to assess prospectively baseline characteristics and outcomes after 12 months (± 90 days) of fingolimod treatment. Patients were divided into 2 cohorts: fingolimod starter [first received fingolimod in PANGAEA (n = 3315)] and previous study [received fingolimod before enrollment in PANGAEA in RCTs (n = 875), some of whom also had baseline data at entry into RCTs (n = 505)]. At PANGAEA baseline, patients in the fingolimod starter versus the previous study cohort had a higher annualized relapse rate [ARR (95% confidence interval): 1.79 (1.75–1.83) vs 1.32 (1.25–1.40)] and Expanded Disability Status Scale score [3.11 (3.04–3.17) vs 2.55 (2.44–2.66)]. A greater proportion in the fingolimod starter versus previous study cohort had diabetes (2.0% vs 0.7%). After 12 months of fingolimod, ARRs were lower than in the 12 months before PANGAEA enrollment in the fingolimod starter [0.386 (0.360–0.414)] and previous study [0.276 (0.238–0.320)] cohorts. Expanded Disability Status Scale scores were stable versus baseline. Adverse events were experienced by similar proportions in both cohorts during fingolimod treatment. Relevant differences exist in disease activity and comorbidities between patients receiving fingolimod in clinical practice versus RCTs. Irrespective of baseline differences indicating a higher proportion at an advanced stage of multiple sclerosis in the real world versus RCTs, fingolimod remains effective, with a manageable safety profile.
Highlights
Randomized controlled trials (RCTs) of disease-modifying therapies (DMTs) in multiple sclerosis (MS) are designed to evaluate treatment efficacy
Such studies are an important follow-on from RCTs, which are often conducted under experimental conditions in a specific population of patients in order to limit the impact of confounding factors when investigating the efficacy of DMTs [6]
The need for real-world studies is illustrated by the present analysis, which used data from the German noninterventional, observational study Post-Authorization Non-interventional German sAfety study of GilEnyA (PANGAEA) to evaluate baseline characteristics and outcomes after 12 months of fingolimod therapy in patients receiving treatment and being monitored according to standard clinical practice
Summary
Randomized controlled trials (RCTs) of disease-modifying therapies (DMTs) in multiple sclerosis (MS) are designed to evaluate treatment efficacy. Robust real-world studies are needed to assess the safety and effectiveness of DMTs in the population of patients being treated in clinical practice, including those with characterized comorbidities and using concomitant medications [4,5,6]. Real-world data can be collected retrospectively from existing data sources, such as MS registries, including the international patient registry MSBase Such registries tend to focus on treatment effectiveness, and provide limited safety information, whereas PANGAEA has been designed to collect robust data for both safety and effectiveness [1, 7,8,9]
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