替代指标的定义演进及评价验证方法介绍

Surrogate endpoint:definition evolution,evaluation and validation

替代指标能够作为有意义的临床结局指标的代替,并可根据流行病学、诊断学、病理学等理论依据来预测干预对终点结局的影响。关于替代指标的评价和验证方法的探讨在国外已有30余年的历史,使用经过验证的替代指标可大大缩减临床试验所需的样本量和研究周期。本研究对替代指标的定义演进、评价和验证方法的原理和应用进行全面介绍,为未来研究中替代指标的使用、评价及验证提供参考。

A surrogate endpoint is intended to substitute for a clinical endpoint and is expected to predict the effect of the intervention on clinical endpoints based on epidemiologic,diagnostic,and pathophysiologic evidence.A validated surrogate endpoint can reduce sample size and follow-up duration of clinical trials;hence,the evaluation and validation methods of surrogate endpoints have been discussed for more than 30 years around the world.This paper comprehensively introduced the definition evolution,evaluation,and validation methods of surrogate endpoints,and provided references for future research.