Presentation (CTI)

OBJECTIVES: The National Healthcare Security Administration of China and the Boao Lecheng International Medical Tourism Pilot Zone (Lecheng) have launched a pilot program utilizing real-world evidence (RWE) to inform drug pricing and reimbursement decision. This study aims to: (1)develop technical guidelines for pharmaceutical companies to apply RWE in evaluating drug effectiveness, safety, and economic value; (2) establish a practical assessment framework enabling independent experts to evaluate RWE quality and provide recommendations to support government reimbursement decision-making.
METHODS: We employed a three-phase approach: (1) Comprehensive literature review of academic papers, policy documents, and HTA RWE guidelines (e.g., UK NICE, Canada CADTH). (2) Delphi consensus process involving focus group discussions and multidisciplinary expert interviews to refine the framework according to China's healthcare security requirements.(3) Mixed-methods validation combining quantitative and qualitative evaluation of RWE quality across nine pharmaceutical case studies.
RESULTS: The study established: 1. A 6-step assessment process: (1) protocol submissionl (2) expert review (3) study optimization (4) data verificationl (5) evidence assessment (6) decision recommendations. 2. Technical guidelines incorporating: (1) A standardized RWS report checklist. (2) A data adequacy self-appraisal worksheet.(3) A tiered assessment checklist (5 core domains, 16 secondary criteria, 40 tertiary items).(4) A qualitative assessment report with structured recommendations across multiple dimensions (clinical effectiveness, safety profile, healthcare resource utilization, and economic value et al.) to support reimbursement decisions.
CONCLUSIONS: The study outcomes have been implemented in China's National Reimbursement Drug List adjustments for 2023 and 2024, with RWE from three drugs successfully supporting reimbursement decision-making. This demonstrates effective integration of international HTA standards with China's regulatory environment through RWE.