N-of-1 Trials, Their Reporting Guidelines, and the Advancement of Open Science Principles.

Harvard data science review Pub Date : 2022-01-01 Epub Date: 2022-09-08 DOI:10.1162/99608f92.a65a257a
Antony Porcino, Sunita Vohra
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引用次数: 2

Abstract

N-of-1 trials are multiple crossover trials done over time within a single person; they can also be done with a series of individuals. Their focus on the individual as the unit of analysis maintains statistical power while accommodating greater differences between patients than most standard clinical trials. This makes them particularly useful in rare diseases, while also being applicable across many health conditions and populations. Best practices recommend the use of reporting guidelines to publish research in a standardized and transparent fashion. N-of-1 trials have the SPIRIT extension for N-of-1 protocols (SPENT) and the CONSORT extension for N-of-1 trials (CENT). Open science is a recent movement focused on making scientific knowledge fully available to anyone, increasing collaboration, and sharing of scientific efforts. Open science goals increase research transparency, rigor, and reproducibility, and reduce research waste. Many organizations and articles focus on specific aspects of open science, for example, open access publishing. Throughout the trajectory of research (idea, development, running a trial, analysis, publication, dissemination, knowledge translation/reflection), many open science ideals are addressed by the individual-focused nature of N-of-1 trials, including issues such as patient perspectives in research development, personalization, and publications, enhanced equity from the broader inclusion criteria possible, and easier remote trials options. However, N-of-1 trials also help us understand areas of caution, such as monitoring of post hoc analyses and the nuances of confidentiality for rare diseases in open data sharing. The N-of-1 reporting guidelines encourage rigor and transparency of N-of-1 considerations for key aspects of the research trajectory.

N-1试验、报告指南和开放科学原则的进展
N-of-1试验是指在同一个人身上进行的多次交叉试验;它们也可以用一系列的个体来完成。与大多数标准临床试验相比,他们将重点放在个体作为分析单位,保持了统计能力,同时适应了患者之间更大的差异。这使得它们对罕见病特别有用,同时也适用于许多健康状况和人群。最佳做法建议使用报告准则,以标准化和透明的方式发表研究成果。N-of-1试验具有用于N-of-1协议(SPENT)的SPIRIT扩展和用于N-of-1试验(CENT)的CONSORT扩展。开放科学是最近的一项运动,其重点是使任何人都能充分获得科学知识,增加合作和分享科学成果。开放科学的目标是提高研究的透明度、严谨性和可重复性,并减少研究浪费。许多组织和文章关注开放科学的特定方面,例如开放获取出版。在研究的整个轨迹中(想法、发展、试验、分析、出版、传播、知识翻译/反思),许多开放科学理想都是通过N-of-1试验的个人关注性质来解决的,包括研究开发中的患者观点、个性化和出版物等问题,从更广泛的纳入标准中增强公平性,以及更容易的远程试验选择。然而,N-of-1试验也有助于我们了解需要谨慎的领域,例如监测事后分析和开放数据共享中罕见疾病保密的细微差别。N-of-1报告准则鼓励对研究轨迹关键方面的N-of-1考虑的严谨性和透明度。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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