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Disease-modifying therapies (DMTs) reduce disease activity and progression in multiple sclerosis (MS), but the DMT landscape has grown increasingly complex as new therapies become available. While randomized studies provide a high level of evidence for DMT efficacy and safety, observational studies harnessing real-world data are being used for direct comparison of DMTs in larger, heterogeneous populations to answer clinical questions with broad applicability.
“The recognized importance of real-world evidence is blossoming,” says neuroimmunologist Carrie M. Hersh, DO, MSc, who serves as Director of the Multiple Sclerosis Health and Wellness Program and Associate Program Director of the Neuroimmunology and MS Fellowship at the Cleveland Clinic Lou Ruvo Center for Brain Health. “Through the work we are implementing at Cleveland Clinic — and others are pursuing around the world — we’ll be able to better personalize care and meet the needs of our patients.”
In the latest episode of Cleveland Clinic’s Neuro Pathways podcast, Dr. Hersh provides insight on the role of observational studies in MS clinical decision-making. She discusses:
- The benefits and limitations of randomized trials compared with observational studies
- The MS Partners Advancing Technology and Health Solutions (MS PATHS) program, a network of 10 MS centers that collect and share patient data for research
- Details of two DMT comparative effectiveness studies
- How observational studies of large patient populations can contribute to individualized care
- What’s next in real-world evidence for MS treatment
Click the podcast player above to listen to the 29-minute episode now, or read on for a short edited excerpt. Check out more Neuro Pathways episodes at clevelandclinic.org/neuropodcast or wherever you get your podcasts.
This activity has been approved for AMA PRA Category 1 Credit™. After listening to the podcast, you can claim your credit here.
Excerpt from the podcast
Dr. Hersh: One of the issues we have to consider when performing a comparative effectiveness study using just one site is that we are only looking at patients at that one site and the information is not generalizable. We also don’t have a systematic way that we are collecting the data, which therefore are not standardized. That might impact the end points on which we are basing conclusions when we’re talking about differences in treatment effects.
To address this, we decided to look at a comparative effectiveness study between these two agents [dimethyl fumarate and fingolimod], but instead of just using data from the EMR through the Cleveland Clinic, we decided to use data from the MS PATHS learning health system. That way, not only were we including a more heterogeneous and larger patient population across multiple sites, but we were also using standardized information, including how the data were collected as part of routine care such as clinical end points, patient-reported outcome measures and MRIs. Additionally, we were able to look at the comparative effects of these two medications on serum neurofilament light chain, an emerging biomarker that is gaining a lot of interest in the MS space as an inflammatory marker and predictor of how patients respond to individual treatments.
The great part is that even though the patient data were different in this study versus the prior Cleveland Clinic study because they came from different sites, we still showed comparability between these two disease-modifying therapies. This is reassuring because it gives us more clarification on how they stack up in clinical practice and shows that the information we get from different study designs and from different data populations is lining up.