Patients and investigators prefer measures of absolute risk in subgroups for pragmatic randomized trials
Patients and investigators prefer measures of absolute risk in subgroups for pragmatic randomized trials
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DOI：
10.1016/j.jclinepi.2018.06.009
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发表时间：
2018-11-01
影响因子：
7.2
通讯作者：
Hernan, Miguel A.
中科院分区：
文献类型：
--
作者：
Murray, Eleanor J.;Caniglia, Ellen C.;Hernan, Miguel A.
目的：实用的随机试验是共同决策的重要工具，但没有指导存在对因果信息类型的患者的偏好。研究设计和设置：我们（a）在马萨诸塞州的波士顿举行了三个焦点小组，患者（n = 23）;（B）调查（n = 12）和访谈（n = 5）具有进行实用性试验经验的研究人员;和（c）进行了系统的文献回顾的实用性试验（n = 63）。结果：患者不信任的新上市的药物，除非大大更有效的比现有的选择，首选分层的绝对风险，并重视依从性调整分析时，他们希望坚持。研究者想要意向治疗和符合方案的效果，但认为缺乏估计符合方案效果的方法。在评估符合方案效应时，许多实用性试验使用了不适当的方法来调整依从性和失访。结论：我们对实用性试验提出了四点建议，以提高以患者为中心：（1）关注有效性或安全性的优越性，而不是非劣效性;（2）让患者参与指定先验亚组;（3）报告风险的绝对测量值;（4）报告风险的绝对测量值。和（4）用有效的符合方案的效应估计值补充意向治疗效应估计值。(C)2018爱思唯尔公司All rights reserved.
Objectives: Pragmatic randomized trials are important tools for shared decision-making, but no guidance exists on patients' preferences for types of causal information. We aimed to assess preferences of patients and investigators toward causal effects in pragmatic randomized trials.Study Design and Setting: We (a) held three focus groups with patients (n = 23) in Boston, MA; (b) surveyed (n = 12) and interviewed (n = 5) investigators with experience conducting pragmatic trials; and (c) conducted a systematic literature review of pragmatic trials (n = 63).Results: Patients were distrustful of new-to-market medications unless substantially more effective than existing choices, preferred stratified absolute risks, and valued adherence-adjusted analyses when they expected to adhere. Investigators wanted both intention-to treat and per-protocol effects but felt methods for estimating per-protocol effects were lacking. When estimating per-protocol effects, many pragmatic trials used inappropriate methods to adjust for adherence and loss to follow-up.Conclusion: We made four recommendations for pragmatic trials to improve patient centeredness: (1) focus on superiority in effectiveness or safety, rather than noninferiority; (2) involve patients in specifying a priori subgroups; (3) report absolute measures of risk; and (4) complement intention-to-treat effect estimates with valid per-protocol effect estimates. (C) 2018 Elsevier Inc. All rights reserved.