PDF(Pubmed)
Abstract：
BACKGROUND: n-of-1 trials are undertaken to optimise the evaluation of health technologies in individual patients. They involve a single patient receiving treatments, both interventional and control, consecutively over set periods of time, the order of which is decided at random. Although n-of-1 trials are undertaken in medical research it could be argued they have the utility to be undertaken more frequently. We undertook the National Institute for Health Research (NIHR) commissioned DIAMOND (Development of generalisable methodology for n-of-1 trials delivery for very low volume treatments) project to develop key points to assist clinicians and researchers in designing and conducting n-of-1 trials.
METHODS: The key points were developed by undertaking a stakeholder workshop, followed by a discussion within the study team and then a stakeholder dissemination and feedback event. The stakeholder workshop sought to gain the perspectives of a variety of stakeholders (including clinicians, researchers and patient representatives) on the design and use of n-of-1 trials. A discussion between the study team was held to reflect on the workshop and draft the key points. Lastly, the stakeholders from the workshop were invited to a dissemination and feedback session where the proposed key points were presented and their feedback gained.
RESULTS: A set of 22 key points were developed based on the insights from the workshop and subsequent discussions. They provide guidance on when an n-of-1 trial might be a viable or appropriate study design and discuss key decisions involved in the design of n-of-1 trials, including determining an appropriate number of treatment periods and cycles, the choice of comparator, recommended approaches to randomisation and blinding, the use of washout periods and approaches to analysis.
CONCLUSIONS: The key points developed in the project will support clinical researchers to understand key considerations when designing n-of-1 trials. It is hoped they will support the wider implementation of the study design.
METHODS: The key points were developed by undertaking a stakeholder workshop, followed by a discussion within the study team and then a stakeholder dissemination and feedback event. The stakeholder workshop sought to gain the perspectives of a variety of stakeholders (including clinicians, researchers and patient representatives) on the design and use of n-of-1 trials. A discussion between the study team was held to reflect on the workshop and draft the key points. Lastly, the stakeholders from the workshop were invited to a dissemination and feedback session where the proposed key points were presented and their feedback gained.
RESULTS: A set of 22 key points were developed based on the insights from the workshop and subsequent discussions. They provide guidance on when an n-of-1 trial might be a viable or appropriate study design and discuss key decisions involved in the design of n-of-1 trials, including determining an appropriate number of treatment periods and cycles, the choice of comparator, recommended approaches to randomisation and blinding, the use of washout periods and approaches to analysis.
CONCLUSIONS: The key points developed in the project will support clinical researchers to understand key considerations when designing n-of-1 trials. It is hoped they will support the wider implementation of the study design.
摘要：
背景：进行n-of-1试验以优化个体患者的健康技术评估。他们涉及一名患者接受治疗，介入和控制，在设定的时间段内连续，其顺序是随机决定的。尽管在医学研究中进行了n-1试验，但可以说它们具有更频繁地进行的效用。我们承担了美国国立卫生研究院（NIHR）委托DIAMOND（开发用于极低量治疗的n-of-1试验的通用方法）项目，以开发关键点，以帮助临床医生和研究人员设计和进行n-of-1试验。
方法：关键点是通过举办利益相关者研讨会来开发的，接下来是研究团队内部的讨论，然后是利益相关者的传播和反馈事件。利益相关者研讨会寻求获得各种利益相关者的观点(包括临床医生，研究人员和患者代表）关于n-of-1试验的设计和使用。研究小组之间进行了讨论，以反思研讨会并起草要点。最后,研讨会的利益攸关方应邀参加了一次传播和反馈会议，会上介绍了拟议的要点，并获得了他们的反馈。
结果：根据研讨会的见解和随后的讨论，制定了一组22个关键点。他们提供关于n-of-1试验何时可能是可行或适当的研究设计的指导，并讨论n-of-1试验设计中涉及的关键决定。包括确定适当的治疗周期和周期数，比较器的选择，推荐的随机化和致盲方法，冲洗期的使用和分析方法。
结论：该项目开发的关键点将支持临床研究人员在设计n-of-1试验时了解关键考虑因素。希望他们将支持研究设计的更广泛实施。
方法：关键点是通过举办利益相关者研讨会来开发的，接下来是研究团队内部的讨论，然后是利益相关者的传播和反馈事件。利益相关者研讨会寻求获得各种利益相关者的观点(包括临床医生，研究人员和患者代表）关于n-of-1试验的设计和使用。研究小组之间进行了讨论，以反思研讨会并起草要点。最后,研讨会的利益攸关方应邀参加了一次传播和反馈会议，会上介绍了拟议的要点，并获得了他们的反馈。
结果：根据研讨会的见解和随后的讨论，制定了一组22个关键点。他们提供关于n-of-1试验何时可能是可行或适当的研究设计的指导，并讨论n-of-1试验设计中涉及的关键决定。包括确定适当的治疗周期和周期数，比较器的选择，推荐的随机化和致盲方法，冲洗期的使用和分析方法。
结论：该项目开发的关键点将支持临床研究人员在设计n-of-1试验时了解关键考虑因素。希望他们将支持研究设计的更广泛实施。
