论文标题
将参与者的福利纳入顺序多次分配随机试验
Incorporating Participants' Welfare into Sequential Multiple Assignment Randomized Trials
论文作者
论文摘要
动态治疗方案(DTR)是决策规则的序列,这些序列根据患者的时间变化临床状况建议治疗。顺序多重分配随机试验(SMART)是一种实验设计,可以为构建最佳DTR提供高质量的证据。在传统的智能中,参与者被随机分配到具有平衡随机概率的多个阶段的可用治疗方法。尽管在比较嵌入式DTR方面具有相对简单的实施和理想的表现,但常规的智能面孔不可避免地道德问题,包括将许多参与者分配给经验上的劣等治疗或他们不喜欢的治疗方法,这可能会减慢招聘程序的速度,并导致较高的磨损率,最终导致较差的内部和外部有效性的试验结果。在这种情况下,我们在实验 - 市场框架(Smart-exam)下提出了一个智能,这是一种新颖的智能设计,通过将他们的偏好和预测的治疗效果纳入随机过程中,具有改善参与者福利的潜力。我们描述了进行智能外观和评估其性能与传统智能相比的步骤。结果表明,智能-EXAM可以改善参加试验的参与者的福利,同时在适当指定实验参数时具有理想的构建最佳DTR的能力。最终,我们使用来自注意力缺陷/多动症(ADHD)的儿童的SMART数据来说明智能外观设计的实际潜力。
Dynamic treatment regimes (DTRs) are sequences of decision rules that recommend treatments based on patients' time-varying clinical conditions. The sequential multiple assignment randomized trial (SMART) is an experimental design that can provide high-quality evidence for constructing optimal DTRs. In a conventional SMART, participants are randomized to available treatments at multiple stages with balanced randomization probabilities. Despite its relative simplicity of implementation and desirable performance in comparing embedded DTRs, the conventional SMART faces inevitable ethical issues including assigning many participants to the empirically inferior treatment or the treatment they dislike, which might slow down the recruitment procedure and lead to higher attrition rates, ultimately leading to poor internal and external validities of the trial results. In this context, we propose a SMART under the Experiment-as-Market framework (SMART-EXAM), a novel SMART design that holds the potential to improve participants' welfare by incorporating their preferences and predicted treatment effects into the randomization procedure. We describe the steps of conducting a SMART-EXAM and evaluate its performance compared to the conventional SMART. The results indicate that the SMART-EXAM can improve the welfare of the participants enrolled in the trial, while also achieving a desirable ability to construct an optimal DTR when the experimental parameters are suitably specified. We finally illustrate the practical potential of the SMART-EXAM design using data from a SMART for children with attention-deficit/hyperactivity disorder (ADHD).