OBJECTIVES: This study aimed to analyze real-world studies (RWS) and their associated real-world data (RWD) sources on central nervous system (CNS) diseases in China.

METHODS: A comprehensive review of RWS from well-established databases published from 2010 to 2022 was conducted, focusing on CNS diseases in the Chinese population. We searched PubMed, Embase, CNKI, and Wanfang databases to identify relevant studies, from which we extracted and synthesized data on RWS characteristics and RWD-source features.

RESULTS: We identified 389 RWS, with 122 in Chinese. There was a notable increase in RWS over the years, from 8 in 2010 to 60 in 2021. Stroke emerged as the most frequently studied CNS disease, featured in 59.6% of the studies. These studies primarily aimed to explore disease epidemiology (59.1%) and clinical effectiveness (10.5%). Notably, 62.4% of RWS had sample sizes above 1000. Geographical analysis revealed that coastal provinces, Beijing, and Sichuan were more commonly represented, likely reflecting regional research strengths. Of the 184 RWD sources identified, registries (64.7%) and electronic medical records (EMR) (22.3%) were predominant. Despite the breadth of hospital networks and participant cohorts, notable data gaps were present, including missing information on the numbers of hospitals (29.3%) and all enrollees (20.1%), and observation periods (28.8%), as these database details were unreported in the RWS.

CONCLUSIONS: The proliferation of RWS for CNS diseases in China signals a trend towards data-driven insights in clinical and policy realms. However, the full potential of RWD is hindered by data deficiencies in completeness, quality, and accessibility. This review’s limitations, such as potential publication bias and the exclusion of non-indexed sources, may limit the findings’ applicability. To enhance the impact of real-world evidence on the understanding and management of CNS diseases, it is critical to improve data integrity and reporting standards.