The n-of-1 clinical trial: the ultimate strategy for individualizing medicine?

被引:456
作者
Lillie, Elizabeth O. [1 ,2 ]
Patay, Bradley [1 ,2 ]
Diamant, Joel [1 ,2 ]
Issell, Brian [1 ,2 ]
Topol, Eric J. [1 ,2 ,3 ,4 ]
Schork, Nicholas J. [1 ,2 ,3 ]
机构
[1] Scripps Hlth, La Jolla, CA 92037 USA
[2] Scripps Translat Sci Inst, La Jolla, CA 92037 USA
[3] Scripps Res Inst, La Jolla, CA 92047 USA
[4] W Wireless Hlth Inst, La Jolla, CA 92037 USA
关键词
clinical equipoise; early-phase trials; individualized medicine; n-of-1 remote phenotyping; single patient trial; treatment repositioning; wireless health; RANDOMIZED CONTROLLED-TRIAL; METHYLPHENIDATE; CHEMOTHERAPY; PARACETAMOL; CHILDREN; IMPROVE; SERIES; DRUGS; METAANALYSIS; EXPERIENCES;
D O I
10.2217/PME.11.7
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
N-of-1 or single subject clinical trials consider an individual patient as the sole unit of observation in a study investigating the efficacy or side-effect profiles of different interventions. The ultimate goal of an n-of-1 trial is to determine the optimal or best intervention for an individual patient using objective data-driven criteria. Such trials can leverage study design and statistical techniques associated with standard population-based clinical trials, including randomization, washout and crossover periods, as well as placebo controls. Despite their obvious appeal and wide use in educational settings, n-of-1 trials have been used sparingly in medical and general clinical settings. We briefly review the history, motivation and design of n-of-1 trials and emphasize the great utility of modern wireless medical monitoring devices in their execution. We ultimately argue that n-of-1 trials demand serious attention among the health research and clinical care communities given the contemporary focus on individualized medicine.
引用
收藏
页码:161 / 173
页数:13
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