目的 回顾患者登记系统的发展过程,总结分析其关键要素,阐述患者登记研究的设计步骤及其在监管决策中的应用,为推动患者登记在我国监管决策中的应用提出合理建议。方法 采用国内外文献研究法,从上市前药械临床评价和上市后药械监测两个方面,明晰患者登记应用时需考虑的关键问题。结果 患者登记可应用于支持监管决策,例如,评估疾病自然史;确定试验或治疗的临床效果,成本效果或相对有效性;衡量或监测与使用特定产品相关的安全性和有效性。结论 某些情况下,将患者登记作为证据开发工具可以填补临床试验结果与实际临床实践之间的空白,但其在我国的应用仍存在短板,因此,加强数据收集和分析的基础设施建设,完善患者登记研究沟通和审评机制至关重要。
Abstract
OBJECTIVE To review the development process of the patient registries, summarize and analyze its key elements and explain the design steps of patient registration research and its application in regulatory decision-making, put forward reasonable suggestions for promoting the application of patient registries in Chinese regulatory decision-making. METHODS Based on domestic and foreign literature research method, from the two aspects of pre-marketing drug device clinical evaluation and post-marketing monitoring, this paper clarified the key issues that need to be considered in patient registries and application. RESULTS Patient registration can be used to support regulatory decision-making, for example, to evaluate the natural history of a disease; determine the clinical effect, cost-effectiveness, or relative effectiveness of a trial or treatment; measure or monitor the safety and effectiveness of specific products. CONCLUSION In some cases, the use of patient registration as an evidence development tool can fill the gap between clinical trial results and actual clinical practice, but its application in China still has shortcomings. Therefore, it is necessary to strengthen the construction of data collection and analysis infrastructure and improve the communication and review mechanism for patient registries studies is crucial.
关键词
患者登记 /
真实世界 /
药械监管
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Key words
patient registry /
real world /
drugs and medical devices regulation
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中图分类号：
R951
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