新药研究项目需要面对Ⅱ期临床研究阶段因有效性原因所致的高失败率。目前受到推崇的解决方案是在早期临床研究中采用机制验证和概念验证的策略。本文介绍了机制验证和概念验证的概念、试验设计要求、重要性等,并给出了研究示例加以说明。期望这种设计思路能够尽快整合到我国自主开发的新药早期临床研究中,为加快新药临床研究项目的推进以及为关键性决策提供依据。 New drug research projects need to face the phase II clinical research stage due to the effectiveness of the reasons for the high failure rate. The currently respected solution is the strategy of using mechanism verification and proof of concept in early clinical studies. This paper introduces the concepts of mechanism verification and proof of concept, test design requirements, importance and so on, and gives examples of research to illustrate. It is hoped that this design idea can be integrated into the early clinical research of new drug developed by our country as soon as possible so as to accelerate the promotion of new drug clinical research project and provide the basis for the key decision-making.