中国循证医学杂志

中国循证医学杂志

真实世界研究与创新药物价值

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随机对照试验(RCTs)是在严格选定的人群里开展的研究,以确保研究结果不受外界各类干扰因素影响并提供有关安全性和有效性的证据信息。真实世界研究是创新技术及药物上市后开展的研究,其通过收集创新技术及药物在临床使用中的实际效果数据,帮助医疗决策者了解新的药物在其被使用的不同医疗环境中所能发挥的真实效果。RCTs 是真实世界研究的基础,真实世界研究是 RCTs 的有益补充。医保数据库是真实世界数据研究的重要数据库之一。目前我国医保正进入新的定位时期,由被动支付和补偿向以价值为基础的战略性购买转变,以期为参保者购买最佳性价比的服务。医保作为民众的医疗基金管理方,需要建立一个成熟、完善、以价值为基础、以临床结果导向为目标的保障机制。医保考虑的核心是价值,是创新药物及技术的性价比而不单一分开地看疗效或价格。评估创新药物价值是医保评估中至关重要的一环。笔者认为创新技术或药物全面价值的评估具体包含临床价值、经济价值、患者价值和社会价值四个维度。

Randomized double-blind controlled trials (RCTs) conduct researches in carefully selected populations to ensure results of RCTs are unaffected by external disturbances and provide evidence of safety and efficacy. Real-world researches further help to understand the real world effects of new technologies in different medical environments after-market authorization. RCTs are the evidence foundation of real-world researches, and real-world researches provide valuable complement to RCTs. Medical insurance database is one of the most important database in real-world researches. Now, China's national medical insurance is entering a new era and transits from passive payment and compensation into a value-based strategic purchase mechanism for its insured population to buy the most cost-effective services. It is necessary to establish a mature, well-organized and value-based mechanism. The core of such mechanism is values, which is the price/performance ratio of innovative medicines and technologies rather than looking at the price solely. Demonstrating innovative drug value is an essential part of health care assessment. The authors argue that the assessment of the overall value of innovative technologies or medicines should include and based on the following four dimensions: clinical value, economic value, patient value and society value.

关键词: 真实世界研究; 随机对照试验; 临床价值; 经济价值; 患者价值; 社会价值

Key words: Real-world research; Randomized controlled trial; Clinical value; Economic value; Patient value; Society value

引用本文: 宣建伟, 李幼平. 真实世界研究与创新药物价值. 中国循证医学杂志, 2018, 18(4): 273-276. doi: 10.7507/1672-2531.201801090 复制

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