Kravitz Richard L, Duan Naihua
Department of Internal Medicine, University of California, Davis.
Sacramento, California, United States of America.
Harv Data Sci Rev. 2022;4(SI3). doi: 10.1162/99608f92.901255e7. Epub 2022 Sep 8.
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.
医学证据发展的主要支柱是平行组随机对照试验（RCT），它能得出试验中普通个体的治疗效果或有效性的估计值。相比之下，个性化试验（有时称为“单人试验”或“N-of-1试验”）评估单一受试者中两种或更多治疗方法的相对有效性。这些单受试者随机交叉试验在医学领域以分散的方式应用了40多年，但尚未得到广泛采用。一个重要障碍是缺乏有力证据表明个性化试验能改善治疗结果。然而，主要障碍可能与疗效证明的关系较小，而更多地与设计和实施的实际方面有关。这些方面包括治疗方案灵活性、盲法和洗脱期的决策，以及组织、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后，本文探讨了这些障碍，并从根本上提出问题：“为什么个性化试验没有得到更广泛的采用，如何使其更容易部署和有用？”文章最后提出，新兴技术和方法有望克服现有障碍，为下一代个性化试验研究人员和从业者开辟充满希望的前景。
