Some design issues in trials of microbicides for the prevention of HIV infection
Some design issues in trials of microbicides for the prevention of HIV infection
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DOI：
10.1086/422603
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发表时间：
2004-08-15
影响因子：
6.4
通讯作者：
Richardson, BA
中科院分区：
文献类型：
--
作者：
Fleming, TR;Richardson, BA
用于评估杀微生物剂的预防人类免疫缺陷病毒（HIV）感染的试验提供了显著的设计挑战。其中三个设计问题值得更仔细的考虑。第一个问题涉及当安慰剂方案可能不是惰性的，并且当干预的有效性在很大程度上取决于行为以及生物因素时，使用盲态和非盲态对照组的益处。第二个问题涉及监管机构批准药物和生物制剂上市所需的证据强度，只有一项关键的3期临床试验提供了此类证据。第三个问题涉及1期试验完成后的适当下一步，以及进行2b期筛选试验的具体优点，这些试验评估了对相同临床疗效终点的影响，这些终点将是3期试验的主要终点。在预防艾滋病毒感染的杀微生物剂试验中考虑的问题在许多其他临床情况下也很重要。
Trials for the prevention of human immunodeficiency virus (HIV) infection that evaluate microbicides provide significant design challenges. Three of these design issues deserve more careful consideration. The first issue relates to the benefits of using both blinded and unblinded control groups when the placebo regimen may not be inert and when the effectiveness of an intervention heavily depends on behavioral, as well as biological, factors. The second issue relates to the strength of evidence required for regulatory approval for the marketing of drugs and biologics when only a single pivotal phase 3 clinical trial has provided such evidence. The third issue relates to the appropriate next step after the completion of phase 1 trials, as well as the specific merits of conducting phase 2b screening trials that assess the effects on the same clinical efficacy end point that will be the primary end point in a phase 3 trial. The issues considered in microbicide trials for the prevention of HIV infection are also of importance in many other clinical scenarios.