PDF(Pubmed)
Abstract：
The mainstay of evidence development in medicine is the parallel-group randomized controlled trial (RCT), which generates estimates of treatment efficacy or effectiveness for the average person in the trial. In contrast, personalized trials (sometimes referred to as \'single-person trials\' or \'N-of-1 trials\') assess the comparative effectiveness of two or more treatments in a single individual. These single-subject, randomized crossover trials have been used in a scattershot fashion in medicine for over 40 years but have not been widely adopted. An important barrier is the paucity of strong evidence that personalized trials improve outcomes. However, the principal impediment may have less to do with proof of efficacy than with practical aspects of design and implementation. These include decisions about treatment regimen flexibility, blinding, and washout periods as well as organizational, clinician, and patient-level challenges. After reviewing the essential elements of personalized trials, this article addresses these speed bumps and fundamentally asks, \'Why have personalized trials not been more widely adopted, and how can they be made more readily deployable and useful?\' The article concludes by suggesting ways in which emerging technologies and approaches promise to overcome existing barriers and open promising vistas for the next generation of personalized-trial researchers and practitioners.
摘要：
医学证据发展的主要是平行组随机对照试验（RCT），对试验中普通人的治疗效果或有效性进行估计。相比之下,个性化试验(有时称为"单人试验"或"N-of-1试验")评估两种或两种以上治疗方法在单个个体中的相对有效性.这些单一主题，40多年来，随机交叉试验一直以分散的方式在医学中使用，但尚未被广泛采用。一个重要的障碍是缺乏强有力的证据表明个性化试验可以改善结果。然而,主要障碍可能与功效证明无关，而与设计和实施的实际方面无关。这些包括关于治疗方案灵活性的决定，盲法,以及清除期和组织期，临床医生,和患者层面的挑战。在回顾了个性化试验的基本要素之后，这篇文章解决了这些减速带，从根本上提出了问题，为什么个性化试验没有得到更广泛的采用，以及如何使它们更易于部署和有用？文章最后提出了新兴技术和方法有望克服现有障碍并为下一代个性化试验研究人员和从业人员打开有希望的前景的方法。
