Over the past decade in China, there has been a substantial increase in real-world evidence (RWE) studies being used to support drug reimbursement and post-marketing decisions. However, due to nuances in the Chinese healthcare system, there are several challenges that must be overcome in order to ensure quality RWE generation.

Possible challenges for registries, chart review studies and electronic medical records (EMR) studies in China include (1) expected loss to follow-up: often patients visit Tier 3 hospitals in the city for initial consultation, diagnosis and treatment initiation. A large proportion of patients then move to Tier 2/Tier 1 hospitals or a rural geographic location for routine visits and continued care or treatment; (2) unstructured data: often data in medical records can be unstructured and primarily contain clinician notes rather than structured medical terminology; (3) fragmented data: data within the hospital setting is often well captured however clinical aspects that take place outside of the hospital (e.g. laboratory assessments, visits to other healthcare professionals, death) will not be integrated into the patient medical records; (4) data access: in addition to ethics committee approval, Human Genetic Resources Administration of China (HGRAC) approval is needed before commencing any study sponsored by a foreign company. Contracting with study sites cannot begin until HGRAC approval is acquired and study timelines must allow for this.

Hybrid design approaches (involving data collection from patient medical records combined with physician/patient reported outcomes) are needed to provide a comprehensive overview of the patient experience and mitigate risks associated with the challenges above. We provide a case example of a technology-based approach (incorporating EMR extraction and electronic PRO [ePRO]), proposed to expedite patient identification and enrolment, ensure quality data capture and allow cost-effective implementation of smaller pilot studies as well as larger retrospective studies or prospective registries.