Harnessing Real-World Data to Inform Decision-Making: Multiple Sclerosis Partners Advancing Technology and Health Solutions (MS PATHS)

Ellen M Mowry, Robert A Bermel, James R Williams, Tammie L S Benzinger, Carl de Moor, Elizabeth Fisher, Carrie M Hersh, Megan H Hyland, Izlem Izbudak, Stephen E Jones, Bernd C Kieseier, Hagen H Kitzler, Lauren Krupp, Yvonne W Lui, Xavier Montalban, Robert T Naismith, Jacqueline A Nicholas, Fabio Pellegrini, Alex Rovira, Maximilian Schulze, Björn Tackenberg, Mar Tintore, Madalina E Tivarus, Tjalf Ziemssen, Richard A Rudick, Ellen M Mowry, Robert A Bermel, James R Williams, Tammie L S Benzinger, Carl de Moor, Elizabeth Fisher, Carrie M Hersh, Megan H Hyland, Izlem Izbudak, Stephen E Jones, Bernd C Kieseier, Hagen H Kitzler, Lauren Krupp, Yvonne W Lui, Xavier Montalban, Robert T Naismith, Jacqueline A Nicholas, Fabio Pellegrini, Alex Rovira, Maximilian Schulze, Björn Tackenberg, Mar Tintore, Madalina E Tivarus, Tjalf Ziemssen, Richard A Rudick

Abstract

Background: Multiple Sclerosis Partners Advancing Technology and Health Solutions (MS PATHS) is the first example of a learning health system in multiple sclerosis (MS). This paper describes the initial implementation of MS PATHS and initial patient characteristics. Methods: MS PATHS is an ongoing initiative conducted in 10 healthcare institutions in three countries, each contributing standardized information acquired during routine care. Institutional participation required the following: active MS patient census of ≥500, at least one Siemens 3T magnetic resonance imaging scanner, and willingness to standardize patient assessments, share standardized data for research, and offer universal enrolment to capture a representative sample. The eligible participants have diagnosis of MS, including clinically isolated syndrome, and consent for sharing pseudonymized data for research. MS PATHS incorporates a self-administered patient assessment tool, the Multiple Sclerosis Performance Test, to collect a structured history, patient-reported outcomes, and quantitative testing of cognition, vision, dexterity, and walking speed. Brain magnetic resonance imaging is acquired using standardized acquisition sequences on Siemens 3T scanners. Quantitative measures of brain volume and lesion load are obtained. Using a separate consent, the patients contribute DNA, RNA, and serum for future research. The clinicians retain complete autonomy in using MS PATHS data in patient care. A shared governance model ensures transparent data and sample access for research. Results: As of August 5, 2019, MS PATHS enrolment included participants (n = 16,568) with broad ranges of disease subtypes, duration, and severity. Overall, 14,643 (88.4%) participants contributed data at one or more time points. The average patient contributed 15.6 person-months of follow-up (95% CI: 15.5-15.8); overall, 166,158 person-months of follow-up have been accumulated. Those with relapsing-remitting MS demonstrated more demographic heterogeneity than the participants in six randomized phase 3 MS treatment trials. Across sites, a significant variation was observed in the follow-up frequency and the patterns of disease-modifying therapy use. Conclusions: Through digital health technology, it is feasible to collect standardized, quantitative, and interpretable data from each patient in busy MS practices, facilitating the merger of research and patient care. This approach holds promise for data-driven clinical decisions and accelerated systematic learning.

Keywords: MS PATHS; digital health technology; learning health system; multiple sclerosis; standardized brain magnetic resonance imaging.

Copyright © 2020 Mowry, Bermel, Williams, Benzinger, de Moor, Fisher, Hersh, Hyland, Izbudak, Jones, Kieseier, Kitzler, Krupp, Lui, Montalban, Naismith, Nicholas, Pellegrini, Rovira, Schulze, Tackenberg, Tintore, Tivarus, Ziemssen and Rudick.

Figures

Figure 1
Figure 1
Probability of completing a follow-up Multiple Sclerosis Performance Test (MSPT; as a function of months between the initial MSPT and completing the second MSPT), (A) overall and by (B) Multiple Sclerosis Partners Advancing Technology and Health Solutions center.
Figure 2
Figure 2
Probability of completing a follow-up standardized magnetic resonance imaging on a Siemens 3T scanner, (A) overall and (B) by individual Multiple Sclerosis Partners Advancing Technology and Health Solutions center.

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