一种新的两阶段疫苗试验设计

AN ALTERNATIVE TWO-STAGE DESIGN OF VACCINE TRIALS

一个设计良好的Ⅱ期疫苗试验,应当同时关注疫苗的有效性和安全性,比如要求疫苗的有效反应率至少达到30%,而不良反应率不超过10%.CONAWAY和PETRONI(1996年)给出一种设计方法,对响应与毒性赋予相同的权重.该文提出新的想法来处理安全性与疗效之间的平衡关系,根据实际需要将反应率和毒性的第一类错误分别确定在不同的显著性水平上;方法较其它设计方案更稳健,而且能控制反应率或毒性的第一类错误.

A good design of phase Ⅱ vaccine trial should focus on both efficacy and safety, for example, requiring the positive response rate above 30% and negative response rate below 10%. CONAWAY and PETRONI proposed a method for designing phase Ⅱ trials that implied an equal weighing of response and toxicity. In this paper, an alternative design was developed to cope with the trade -off between safety and efficacy, which controlled for the marginal type Ⅰ errors of response rate and toxicity rate separately. The proposed method has advantage over other designs that it could definitely control type Ⅰ error of the interests such as treatment antitumor activity or safety and is robust against the real association parameter.