Leveraging Real-World Evidence and Observational Studies in Treating Multiple Sclerosis

Randomized controlled trials (RCTs) are essential for regulatory approval of disease-modifying therapies (DMTs), yet their strict selection criteria often lead to limited generalizability. Observational studies using real-world data (RWD) allow for more inclusive heterogeneous cohorts resulting in higher external validity to inform treatment practices. As reviewed in this article, well-designed comparative effectiveness studies are an important application of RWD. Although, like RCTs, observational studies have their own set of limitations, including various biases that may confound results, advanced statistical methods can mitigate many of these limitations. A focus on personalized treatment will continue to add value to individualize MS care.