真实世界研究的方法与实践

Method and Practice of Real World Study

随机对照临床试验是目前获得证据、制定诊疗策略的主要依据之一,但是,随机对照试验并不总能代表临床实践中的真实情况。真实世界研究是观察性研究,通过"真实世界样本"来反映真实世界总体。临床试验关注效力研究,而真实世界研究则关注效果研究。真实世界研究需要使用多重倾向性评分来减少协变量在组间分布的不均衡性。近年来,真实世界研究正越来越多地应用到临床研究中,可将经验医学与循证医学结合起来。临床试验和真实世界研究是承启关系,前者的结果需要后者的进一步验证及拓展,二者综合考虑才是最佳选择。

Randomized controlled trial （RCT） is so far considered as an important way to provide evidence to make up therapeutic strategy. However, sometimes it is limited to represent the authentic condition during clinical practice. Whereas, Real world study （RWS）, a kind of observational studies, is applied to present the real world population through analysis of ＂real world sample＂. RCT pays more attention to efficacy trials. While RWS more concentrates on effectiveness trials. RWS requires giving propensity scores to decrease covariant malconformation among groups. In the past few years, RWS has been increasingly applied in clinical studies to integrate experience medicine with evidence-based medicine. Results of RCT need to be tested and expanded forward in real world study. Therefore, comprehensive evaluations by both RCT and RWS are supposed to be the best selection.