IS CHINA LEADING IN RWE? A SCOPING REVIEW OF RWE GENERATED FROM CHINA HAINAN BOAO LECHENG PILOT ZONE
Author(s)
YUCHEN GE, MSc1, QIAN ZHAO, BS, MS2, Sijuan Zhou, MS3;
1Bridge Value & Access, Toronto, ON, Canada, 2Department of Health Sciences, University of York, York, United Kingdom, 3The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China
1Bridge Value & Access, Toronto, ON, Canada, 2Department of Health Sciences, University of York, York, United Kingdom, 3The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China
OBJECTIVES: Since its implementation in 2019, the Hainan Boao Lecheng International Medical Tourism Pilot Zone (Pilot Zone) has enabled early clinical use of innovative drugs and medical devices in China and facilitated the generation of real-world evidence (RWE) to support regulatory decision-making. This study aims to systematically evaluate the characteristics and quality of RWE generated from the Pilot Zone.
METHODS: A targeted literature search was conducted in MEDLINE, Cochrane Library, ClinicalTrials.gov, Chinese database Yiigle and Wanfang. Eligible studies included real-world studies (RWS) of innovative health technologies conducted in the Pilot Zone and published in English or Chinese since 2019. Study characteristics were extracted. Market approval and national reimbursement status were confirmed through separate searches.
RESULTS: Sixteen studies evaluating 15 health technologies (five medical devices and ten drugs) were identified across predominantly chronic disease areas, including ophthalmology (n=3), oncology (n=4), inflammatory skin disorders (n=2), cardiometabolic diseases (n=2), and single studies in chronic graft-versus-host disease, Alzheimer’s disease, hearing loss and urologic disorder. Study sample size ranged from 20 to 996. Seven studies were retrospective, six prospective; one ambispective and two incorporated external control arms. Study design included cohort, case series, target value control, chart reviews, and case-only studies. Electronic medical records and follow-up were the primary data sources. Most analyses were descriptive; only two conducted multivariate analyses, and one applied inverse probability of treatment weighting to address confounding. Five drugs have subsequently been included in the national reimbursement drug list.
CONCLUSIONS: Despite supporting accelerated market approvals, RWE from the Pilot Zone remains constrained by small sample sizes, short follow-up periods, and limited representativeness of the national population. The scarcity of RWS on treatments for rare diseases warrants greater attention. Future studies should leverage longer follow-up data and apply more robust analytical methods to support causal inference and confounding control.
METHODS: A targeted literature search was conducted in MEDLINE, Cochrane Library, ClinicalTrials.gov, Chinese database Yiigle and Wanfang. Eligible studies included real-world studies (RWS) of innovative health technologies conducted in the Pilot Zone and published in English or Chinese since 2019. Study characteristics were extracted. Market approval and national reimbursement status were confirmed through separate searches.
RESULTS: Sixteen studies evaluating 15 health technologies (five medical devices and ten drugs) were identified across predominantly chronic disease areas, including ophthalmology (n=3), oncology (n=4), inflammatory skin disorders (n=2), cardiometabolic diseases (n=2), and single studies in chronic graft-versus-host disease, Alzheimer’s disease, hearing loss and urologic disorder. Study sample size ranged from 20 to 996. Seven studies were retrospective, six prospective; one ambispective and two incorporated external control arms. Study design included cohort, case series, target value control, chart reviews, and case-only studies. Electronic medical records and follow-up were the primary data sources. Most analyses were descriptive; only two conducted multivariate analyses, and one applied inverse probability of treatment weighting to address confounding. Five drugs have subsequently been included in the national reimbursement drug list.
CONCLUSIONS: Despite supporting accelerated market approvals, RWE from the Pilot Zone remains constrained by small sample sizes, short follow-up periods, and limited representativeness of the national population. The scarcity of RWS on treatments for rare diseases warrants greater attention. Future studies should leverage longer follow-up data and apply more robust analytical methods to support causal inference and confounding control.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR111
Topic
Health Policy & Regulatory
Disease
SDC: Diabetes/Endocrine/Metabolic Disorders (including obesity), SDC: Oncology, SDC: Sensory System Disorders (Ear, Eye, Dental, Skin), SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain), STA: Multiple/Other Specialized Treatments