Abstract: 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.

Key words: innovative drugs, exploratory investigational new drug, phase 0 clinical trial