Senn Stephen
Competence Centre for Methodology and Statistics, Luxembourg Institute of Health, L-1445, Strassen, Luxembourg.
Stat Med. 2016 Mar 30;35(7):966-77. doi: 10.1002/sim.6739. Epub 2015 Sep 28.
Various sources of variation in observed response in clinical trials and clinical practice are considered, and ways in which the corresponding components of variation might be estimated are discussed. Although the issues have been generally well-covered in the statistical literature, they seem to be poorly understood in the medical literature and even the statistical literature occasionally shows some confusion. To increase understanding and communication, some simple graphical approaches to illustrating issues are proposed. It is also suggested that reducing variation in medical practice might make as big a contribution to improving health outcome as personalising its delivery according to the patient. It is concluded that the common belief that there is a strong personal element in response to treatment is not based on sound statistical evidence.
本文考虑了临床试验和临床实践中观察到的反应存在的各种变异来源，并讨论了估计相应变异成分的方法。尽管这些问题在统计文献中通常已有充分阐述，但在医学文献中似乎理解不足，甚至统计文献偶尔也会出现一些混淆。为了增进理解和交流，本文提出了一些简单的图形方法来说明这些问题。本文还指出，减少医疗实践中的变异可能对改善健康结果做出与根据患者情况个性化治疗同样大的贡献。本文得出结论，认为治疗反应中存在强烈个人因素的普遍观点并非基于可靠的统计证据。
