创新药物的零期临床试验

为了引导创新药物的快速开发、控制新药研发过程中的临床风险，美国食品和药物管理局于2006年颁布了“探索性新药研究”指导原则，提出在进行传统的Ⅰ期临床试验之前开展零期临床试验的概念，并取得了一系列有意义的结果。随着我国自主创新药物的研发体系的建立和发展，能够快速筛选、降低成本、减少风险、提高新药开发效率的探索性新药临床研究方法愈来愈受到关注。但在我国零期临床研究尚处于起步阶段，没有相应的法规和指导原则，缺乏合理的研究设计和专业的研究人员，零期临床研究本身也存在一些局限。本文就创新药物的零期临床试验的概念、研究方法、检测方法、适用药物、优势与不足及与传统临床试验的区别等做一综述。

Phase 0 Clinical Trial of Innovative Drugs

In order to expedite development and reduce risk of drug innovations, the United States Food and Drug Administration （FDA） published “The Exploratory Investigational New Drug Guidance” in 2006 and introduced the concept of early clinical studies prior to traditional Phase I clinical trials, also known as Phase 0 clinical trials. This study model has shown certain advantages in clinical trials conducted in the United States. With the development of innovative drugs in China, new investigational models intended to enhance the drug development process by reducing risk and cost are receiving growing attention. However, Phase 0 clinical trials are still in its nascence in China and lack sufficient government guidance, trial design and experienced research personnel. In this paper, we will discuss the benefits and disadvantages of Phase 0 clinical trials through close analyses of its concept, research methods, assessment, suitable drug types, and differences from traditional clinical trials.