OBJECTIVES: As legislation and incentives to advance the sharing of health data in China increase over time, we aimed to investigate the trends and potentials of real-world data (RWD) sources in oncology and rare diseases in China.

METHODS: Databases containing Chinese population were identified through systematic literature review of real-world studies (RWS). Articles in oncology and rare diseases published from January 2010 to August 2022 in PubMed/Medline, EMBASE, China National Knowledge Infrastructure, and Wanfang were extracted. We summarized characteristics of these RWS and databases used. The databases were categorized into registries, claims, regional electronic health record (rEHR), and health care data aggregators (HCDA).

RESULTS: We identified 446 English and 492 Chinese RWS articles, of which 90.5% were in oncology and 94.6% were peer-reviewed. The number of RWS increased over years in oncology (from 27 in 2010 to ≥105/year after 2019) and rare diseases (from 1 in 2010 to >10/year after 2020). The proportions of RWS in oncology evaluating disease epidemiology and natural history, clinical effectiveness/safety, treatment patterns/outcomes, and health economics after 2017 were 47.3%, 11.0%, 29.7%, and 4.5% respectively. The corresponding proportions in rare diseases were 69.6%, 6.5%, 15.2%, and 13.0%. In oncology, 238 registries, 14 claims, 3 rEHR, and 2 HCDA databases were identified, of which 18.1% containing ≥100,000 participants, 28.4% covering ≥10 hospitals and 44.6% having ≥10 years of follow-up. In rare diseases, 42 registries, 4 claims, and 2 rEHR databases were identified, of which 18.9% containing ≥1,000 participants, 24.5% covering ≥10 hospitals and 26.4% having ≥10 years of follow-up.

CONCLUSIONS: The number of RWD sources is increasing rapidly in China. Data application requires consideration in evolving academic guidance and quality assurance of RWD. Consistent regulatory procedures for database access and partnership across healthcare eco-system are essential for promoting database aggregation and unlocking values of real-world evidence.