使用工具变量在受未测量混杂因素影响的盲态和非盲态随机临床试验中区分治疗和安慰剂效应
Using instrumental variables to disentangle treatment and placebo effects in blinded and unblinded randomized clinical trials influenced by unmeasured confounders
摘要:
临床试验传统上采用盲法作为设计机制,以减少安慰剂效应的影响。然而,在实践中,很难或不可能对研究参与者进行盲态试验,而非盲态试验在医学研究中很常见。在这里,我们展示了如何工具变量可以用来量化和解开治疗和安慰剂的效果在随机临床试验中比较对照和活性治疗的存在混杂因素。其关键思想是使用随机化来分别操纵治疗分配和心理鼓励信息,以增加参与者对改善症状的渴望。所提出的方法能够改善盲法研究中处理效应的估计,最重要的是,打开了大门,以解释非盲法试验中的安慰剂效应。
英文摘要:
Clinical trials traditionally employ blinding as a design mechanism to reduce the influence of placebo effects. In practice, however, it can be difficult or impossible to blind study participants and unblinded trials are common in medical research. Here we show how instrumental variables can be used to quantify and disentangle treatment and placebo effects in randomized clinical trials comparing control and active treatments in the presence of confounders. The key idea is to use randomization to separately manipulate treatment assignment and psychological encouragement messages that increase the participants' desire for improved symptoms. The proposed approach is able to improve the estimation of treatment effects in blinded studies and, most importantly, opens the doors to account for placebo effects in unblinded trials.
机构、总结、中文摘要均由AI分析而来,可能存在错误,请以论文正文为准。