Neuhauser Duncan V, Chu Jennifer
Department of Epidemiology and Biostatistic, School of Medicine, Case Western Reserve University, Cleveland, Ohio, USA.
Department of Physical Medicine and Rehabilitation, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
BMJ Innov. 2017 Jul;3(3):157-162. doi: 10.1136/bmjinnov-2016-000177. Epub 2017 Sep 16.
Randomised clinical trials are designed to determine whether a particular treatment is appropriate to make a significant difference to the health of a defined population and to aid its approval for use. For an accurate, cheap and simple assessment to see if a treatment benefits an individual person, all that is needed is a pen, paper, simple pocket calculator and daily recording of a few variables. It requires the ability to read and write and to understand addition and division. Factorial design of experiments is used to show the impact of several variables and their interaction on the person's health status. An example of a 75-year-old man with an enlarged prostate is used here to illustrate this approach. This person was able to understand and reduce side effects, lower the costs of medication by 83% and improve measured health status by 28%. A multivariate model for this person was then created with about 450 person-days of data.
随机临床试验旨在确定某种特定治疗方法是否适合对特定人群的健康产生显著影响，并有助于其获批使用。要进行准确、廉价且简单的评估，以判断一种治疗方法是否对个体有益，所需要的只是一支笔、一张纸、一个简单的袖珍计算器以及对几个变量的每日记录。这需要具备读写能力以及理解加法和除法的能力。实验的析因设计用于展示几个变量及其相互作用对个人健康状况的影响。此处以一名75岁前列腺肿大的男性为例来说明这种方法。此人能够理解并减少副作用，将药物成本降低83%，并将测量的健康状况提高28%。然后利用约450人日的数据为此人创建了一个多变量模型。
