基于有效性及伦理学原则探讨新药Ⅱ、Ⅲ期临床试验的设计思路与方法

Design idea and methodology for the Ⅱ and Ⅲ clinical trial of the new drugs in view of effectiveness and ethics

基于新药临床研究中选择阳性药物为对照存在可能无法真实评价新药疗效的可能性,以及新药临床试验本身存在的广义伦理学问题,提出新药临床研究过程中,至少应在Ⅱ期选择安慰剂作为对照组,视研究目标疾病情况可以选择病情较轻的受试者参加试验,在首先验证新药的绝对有效性的基础上安排Ⅱ期二阶段和Ⅲ期临床试验:Ⅱ期二阶段可以采用add-on研究模式,适当扩大适应征;Ⅲ期临床试验可以选择安慰剂,也可以选择合适的阳性药物作为对照组,从而既保证了新药的绝对有效性,又使新药临床试验更加符合伦理学原则。

For clinical trials,efficacy evaluation of new drugs might not be reliable if it is based on positive drugs for comparison.Furthermore,the clinical trial itself might encounter general ethical problems.When proposing clinical trials for new drugs,placebo drugs should be chosen for comparison during Phase II.In view of research objective and disease situation,patients with mild conditions should be chosen for clinical trials.On the basis of effectiveness of the new drugs,second stage of Phase II and Phase III can be arranged.Add-on research methodology can be adopted in the second stage of Phase II to adequately extend indication.Phase III clinical trials can choose placebo or suitable positive drugs for comparison.This will ensure the effectiveness of the new drugs as well as meet the ethical requirements.