Leveraging real-world data to investigate multiple sclerosis disease behavior, prognosis, and treatment

• Published in: Multiple sclerosis Vol. 26; no. 1; pp. 23 - 37
• Main Authors: Cohen, Jeffrey A, Trojano, Maria, Mowry, Ellen M, Uitdehaag, Bernard MJ, Reingold, Stephen C, Marrie, Ruth Ann
• Format: Journal Article
• Language: English
• Published: London, England SAGE Publications 01.01.2020
• Subjects: Humans; Information Storage and Retrieval - methods; Information Storage and Retrieval - standards; Multiple Sclerosis - diagnosis; Multiple Sclerosis - therapy; Observational Studies as Topic - methods; Observational Studies as Topic - standards; Randomized Controlled Trials as Topic - methods; Randomized Controlled Trials as Topic - standards; Multiple sclerosis; real-world data; pragmatic clinical trials; real-world evidence; observational studies
• ISSN: 1352-4585; 1477-0970
• DOI: 10.1177/1352458519892555

Randomized controlled clinical trials and real-world observational studies provide complementary information but with different validity. Some clinical questions (disease behavior, prognosis, validation of outcome measures, comparative effectiveness, and long-term safety of therapies) are often better addressed using real-world data reflecting larger, more representative populations. Integration of disease history, clinician-reported outcomes, performance tests, and patient-reported outcome measures during patient encounters; imaging and biospecimen analyses; and data from wearable devices increase dataset utility. However, observational studies utilizing these data are susceptible to many potential sources of bias, creating barriers to acceptance by regulatory agencies and the medical community. Therefore, data standardization and validation within datasets, harmonization across datasets, and application of appropriate analysis methods are important considerations. We review approaches to improve the scope, quality, and analyses of real-world data to advance understanding of multiple sclerosis and its treatment, as an example of opportunities to better support patient care and research.