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

Albert Aboseif; Izanne Roos; Stephen Krieger; Tomas Kalincik; Carrie M Hersh

doi:10.1016/j.ncl.2023.06.003

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

Neurol Clin. 2024 Feb;42(1):203-227. doi: 10.1016/j.ncl.2023.06.003. Epub 2023 Jul 21.

Authors

Albert Aboseif¹, Izanne Roos², Stephen Krieger³, Tomas Kalincik⁴, Carrie M Hersh⁵

Affiliations

¹ Department of Neurology, Neurological Institute, Cleveland Clinic, 9500 Euclid Avenue S10, Cleveland, OH 44195, USA.
² Department of Neurology, Neuroimmunology Centre, Royal Melbourne Hospital, L7 635 Elizabeth Street, Melbourne 3000, Australia; Department of Medicine, CORe, University of Melbourne, Melbourne, Australia.
³ Corinne Goldsmith Dickinson Center for MS Icahn School of Medicine at Mount Sinai, 5 East 98th Street, Box 1138, New York, NY 10029, USA.
⁴ Department of Medicine, CORe, University of Melbourne, Melbourne, Australia; Department of Neurology, Neuroimmunology Centre, Royal Melbourne Hospital, L6 635 Elizabeth Street, Melbourne 3000, Australia.
⁵ Lou Ruvo Center for Brain Health, Cleveland Clinic, 888 West Bonneville Avenue, Las Vegas, NV 89106, USA. Electronic address: hershc@ccf.org.

PMID: 37980116
DOI: 10.1016/j.ncl.2023.06.003

Abstract

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.

Keywords: Disease-modifying therapies; Multiple sclerosis; Observational studies; Propensity score analysis; Randomized controlled trials; Real-world evidence.

Publication types

Review

MeSH terms

Humans
Multiple Sclerosis* / drug therapy
Observational Studies as Topic
Patient Selection