Chen Connie E, Dhruva Sanket S, Bero Lisa A, Redberg Rita F
School of Medicine, University of California, San Francisco, San Francisco, CA 94143-0124, USA.
Arch Intern Med. 2011 Mar 28;171(6):534-9. doi: 10.1001/archinternmed.2010.445. Epub 2010 Nov 22.
Training patients are the first individuals in whom a physician uses an investigational device. There is great variability in the use of data from training patients in the absence of guidelines. The prevalence and extent of data reporting from training patients in cardiovascular device studies submitted for US Food and Drug Administration (FDA) approval has not been characterized.
Information on training patients was abstracted from the Summary of Safety and Effectiveness Data summarizing cardiovascular device premarket applications approved by the FDA from 2000 through 2007. We examined the numbers and characteristics of training patients and the inclusion of their results in end-point analyses.
There were 78 cardiovascular device summaries in this 8-year period, of which 17 (22%) involved training patients. Of the 123 studies in the summaries, 20 (16%) used training patients. All studies excluded training patients from efficacy analyses and 19 of 20 (95%) excluded them from safety analyses. Sixteen of 20 (80%) did not provide any outcome data, and 15 of 20 (75%) did not check for outcome differences between training and nontraining treatment patients. Eighteen of 20 (90%) did not provide demographic information on training patients, and 14 of 20 (70%) did not prespecify guidelines for their enrollment.
Training patients comprise a considerable proportion of patients receiving investigational cardiovascular devices, but their results are excluded from FDA submissions. Their exclusion from analyses means that safety and efficacy outcomes may look better than actual results. Guidelines on the use and inclusion of results for training patients would improve accuracy on results reporting.
试验患者是医生首次使用研究性器械的对象。在缺乏指南的情况下，试验患者数据的使用存在很大差异。提交给美国食品药品监督管理局（FDA）批准的心血管器械研究中，试验患者数据报告的普遍性和程度尚未得到描述。
从2000年至2007年FDA批准的心血管器械上市前申请的安全性和有效性数据摘要中提取试验患者的信息。我们检查了试验患者的数量和特征，以及他们的结果是否纳入终点分析。
在这8年期间有78份心血管器械摘要，其中17份（22%）涉及试验患者。在这些摘要中的123项研究中，20项（16%）使用了试验患者。所有研究都将试验患者排除在疗效分析之外，20项研究中有19项（95%）将他们排除在安全性分析之外。20项研究中有16项（80%）未提供任何结局数据，20项研究中有15项（75%）未检查试验患者与非试验患者治疗结局的差异。20项研究中有18项（90%）未提供试验患者的人口统计学信息，20项研究中有14项（70%）未预先指定其入选标准。
试验患者占接受研究性心血管器械治疗患者的相当比例，但他们的结果被排除在FDA申报材料之外。将他们排除在分析之外意味着安全性和有效性结局可能看起来比实际结果更好。关于试验患者数据使用和结果纳入的指南将提高结果报告的准确性。
