Objective: Standardizing Clinical Outcome Assessments (COAs) in clinical trials and the integration of Real-World Evidence (RWE) and Patient Experience Data into the approval process are 2 key features which are currently being implemented by the FDA (21^st Century Cures Act). In Relapse Remitting Multiple Sclerosis (RRMS) the Expanded Disability Status Scale (EDSS) COA has been fully integrated, however, integration of other COAs important to patients, such as quality of life, neuropsychiatric and neurological symptoms and productivity are less established. We aimed to map out the COAs used in RRMS RWE studies (RWS) and investigate the integration of COAs in RWS over time.

Methods: A literature search was performed using EMBASE to identify original articles from 2000–Dec 20, 2019, reporting on RRMS. We identified RWS publications using a search string based on previously published ISPOR recommendations. Descriptive statistics were used to summarize the findings.

Results: 1,085 original RWS were identified. Most studies included medical records (67.6%), were retrospective (52.0%) and were non-interventional (54.9%). The number of RWS increased 7-fold in the last decade (24 articles in 2009 and 169 articles in 2019). Any COA was reported in 55.4% (n=601) of RWS. Similar to the increase in RWS, studies reporting any COA showed an 8-fold increase in the last decade (11 vs 92 articles). Over 200 different COAs were identified, with EDSS being the most prevalent (65.2%, n=392). Other COAs identified in RWS included quality of life (9.0%), cognition (11.6%), neuropsychiatric symptoms (14.1%) and productivity (5.8%).

Conclusions: The increase in both total RWS and subsequent reporting of COAs in RRMS reflects the increasing need and transparency for RWE and COAs to stakeholders. However, despite the relatively good integration of EDSS in RWE reporting, COAs reporting on other aspects of RRMS important to the patient experience is still poorly integrated and diverse.