OBJECTIVES: Generating real world evidence (RWE) from prospective longitudinal studies like registries can provide critical data to demonstrate safety and efficacy, especially long-term, of MS treatments. The objective is to review US registries for MS, as potential RWD sources for collaborative research to assess the clinical effectiveness of MS therapies.

METHODS: A targeted literature search was conducted November 2020 with no date limitations. Information on study start and completion dates, study status, age, sample size and sponsor was extracted in MS Excel. Results were descriptive.

RESULTS: Forty US registries were identified from the search. Study start dates ranged from 1996 to 2019; 42.5% initiated between 2000-2010 (17/40) and 45% after 2010 (18/40). Registries initiated after 2015 included the PREG-MS, NARCRMS, Corrona, and the VA MS Surveillance Registry. Thirteen registries were completed (32.5%), and more than one-half (55%; 22/40) were active or ongoing. Patient recruitment ranged from 10 patients to 100,000 patients: 42.5% (17/40) had recruitment levels of unknown or below 1000 patients, 40% (16/40) had recruitment targets between 1000-10,000 patients, and 12.5% (5/40) had recruitment levels >10,000 patients. Twenty-percent (8/40) were pregnancy registries; half were currently enrolling patients. Study outcomes included disease progression, adverse events, clinical outcomes like the Expanded Disability Status Scale (EDSS), and patient-reported outcomes assessing health-related quality of life (HRQoL) like the SF-12, MS Impact Scale (MSIS) and Neuro-QoL Adult Short Form (ASF). Less than one-half (42.5%) of the registries were industry funded.

CONCLUSIONS: As new therapies for MS enter the US market, there is an increasing demand for manufacturers to provide RWE to demonstrate long-term safety and efficacy of their medicines. Unlike other diseases, national organizations have invested in registries for MS, providing opportunities for potential collaboration and cost-effective options for researchers and manufacturers.