Abstract: National regulatory authorities and experts have published a series of guidelines and studies on real-world data and real-world evidence, but there is still no clear document guidance on the detailed protocol and statistical analysis methodology of real-world research. We comprehensively reviewed various documents and studies related to real-world data, based on the experience of research protocol design, and developed the research design key points and statistical analysis based on real-world data. The protocol of real-world research needs to explain the applicability of real-world data, comprehensively explain data governance methods from the aspects of data source, data relevance, data integrity, data accuracy and data extraction methods, and evaluate data quality. Statistical analysis methods are used to analyze the reliability and robustness of real-world research conclusions, thus to produce high-quality real-world evidence.

Key words: Real-world study; Protocol design; Key point of design; Statistical analysis

摘要： 国家监管部门及研究领域专家发表了针对真实世界数据和真实世界证据的一系列指导原则和研究，但真实世界研究的详细撰写方法和设计分析要点都仍未有明确的文件指导。本文综合梳理真实世界数据相关的各项文件和研究，结合研究方案设计经验，形成基于真实世界数据研究的设计要点和统计分析解析。真实世界研究的研究方案中需要充分说明真实世界数据的适用性，从数据来源、数据相关性、数据完整性、数据准确性和数据提取方法等方面全面阐述数据治理方法，评估数据质量。利用统计学方法分析真实世界研究结论的可靠性和稳健性，得出高质量的真实世界证据。

关键词: 真实世界研究; 方案设计；设计要点；统计分析