Characterizing Real-World Evidence Pathways in China: Policy Evolution, the Boao Lecheng Pilot, and Implications for Regulatory and Reimbursement Decisions
Author(s)
Lisa Sun, Master1, Hannah Jin, Bachelor2.
1RWE, Tigermed, Shanghai, China, 2BD, Tigerise, Tokyo, Japan.
1RWE, Tigermed, Shanghai, China, 2BD, Tigerise, Tokyo, Japan.
OBJECTIVES: China has introduced multiple initiatives to integrate RWE into regulatory and reimbursement decision-making. These include national-level guidance and the Boao Pilot Zone, which enables early access to globally approved therapies and supports localized RWE generation. However, the role and influence of RWE across different submission pathways remain under-characterized. Objective: To characterize China’s RWE landscape by (1) mapping policy developments, (2) comparing RWE use in Boao and general submissions, (3) evaluating the role and influence of RWE in regulatory and reimbursement decisions.
METHODS: A structured case assessment was conducted for RWE use in regulatory approvals and NRDL listings from 2019 to 2025. Publicly available sources were reviewed, including NMPA announcements, NRDL negotiation outcomes, Boao pilot updates, published literature, and manufacturer or media reports. Cases were included if RWE use was explicitly cited or confirmed through multiple public sources. For each case, we captured: 1) whether RWE was used, 2) RWE type (e.g., effectiveness, external control, safety, adherence, PROs), 3) how much it influenced the decision, scored from 0 (not mentioned) to 4 (pivotal).
We summarized patterns using descriptive comparisons and reviewed policy milestones to assess how RWE use evolved over time.
RESULTS: As of February 2025, over 40 products had entered the Boao RWE pilot, with 21 receiving NMPA approval. Three drugs (Fluocinolone acetonide, Trilaciclib, and Belumosudil Mesylate) were listed in the NRDL based on Boao-generated RWE. These submissions used prospective data and showed higher average impact scores (3-4). In contrast, general pathway submissions relied on retrospective data, and due to limited public disclosure, the number and depth remain unclear.
CONCLUSIONS: China’s RWE strategy reflects a dual approach, combining top-down policy development with regionally driven pilot innovation. The Boao model offers a structured pathway for generating and applying RWE in regulatory and reimbursement. Improved transparency could strengthen future assessments and inform global RWE implementation.
METHODS: A structured case assessment was conducted for RWE use in regulatory approvals and NRDL listings from 2019 to 2025. Publicly available sources were reviewed, including NMPA announcements, NRDL negotiation outcomes, Boao pilot updates, published literature, and manufacturer or media reports. Cases were included if RWE use was explicitly cited or confirmed through multiple public sources. For each case, we captured: 1) whether RWE was used, 2) RWE type (e.g., effectiveness, external control, safety, adherence, PROs), 3) how much it influenced the decision, scored from 0 (not mentioned) to 4 (pivotal).
We summarized patterns using descriptive comparisons and reviewed policy milestones to assess how RWE use evolved over time.
RESULTS: As of February 2025, over 40 products had entered the Boao RWE pilot, with 21 receiving NMPA approval. Three drugs (Fluocinolone acetonide, Trilaciclib, and Belumosudil Mesylate) were listed in the NRDL based on Boao-generated RWE. These submissions used prospective data and showed higher average impact scores (3-4). In contrast, general pathway submissions relied on retrospective data, and due to limited public disclosure, the number and depth remain unclear.
CONCLUSIONS: China’s RWE strategy reflects a dual approach, combining top-down policy development with regionally driven pilot innovation. The Boao model offers a structured pathway for generating and applying RWE in regulatory and reimbursement. Improved transparency could strengthen future assessments and inform global RWE implementation.
Conference/Value in Health Info
2025-09, ISPOR Real-World Evidence Summit 2025, Tokyo, Japan
Value in Health Regional, Volume 49S (September 2025)
Code
RWD100
Topic Subcategory
Health & Insurance Records Systems
Disease
No Additional Disease & Conditions/Specialized Treatment Areas