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金标准是什么?为随机对照试验辩护。

What's in a gold standard? In defence of randomised controlled trials.

作者信息

Backmann Marius

机构信息

Konstanz University, Constance, Germany.

出版信息

Med Health Care Philos. 2017 Dec;20(4):513-523. doi: 10.1007/s11019-017-9773-2.

DOI:10.1007/s11019-017-9773-2
PMID:28432483
Abstract

The standardised randomised clinical trial (RCT) has been exceedingly popular in medical research, economics, and practical policy making. Recently, RCTs have faced criticism. First, it has been argued by John Worrall that we cannot be certain that our sample is not atypical with regard to possible confounding factors. I will argue that at least in the case of medical research, we know enough about the relevant causal mechanisms to be justified to ignore a number of factors we have good reason not to expect to be disruptive. I will also argue against an argument provided by Nancy Cartwright and Eileen Munro that RCTs should not be taken to deductively infer probabilistic causal claims, but ampliatively. The paper will end on a discussion of evidence hierarchies and a defence of the stance of evidence-based medicine that RCTs are the best available method to assess a treatment's efficacy.

摘要

标准化随机临床试验(RCT)在医学研究、经济学和实际政策制定中极为流行。最近,RCT面临批评。首先,约翰·沃拉尔认为,我们无法确定我们的样本在可能的混杂因素方面并非非典型。我将论证,至少在医学研究的情况下,我们对相关因果机制了解得足够多,有理由忽略一些我们有充分理由认为不会造成干扰的因素。我还将反驳南希·卡特赖特和艾琳·芒罗提出的一个观点,即不应将RCT视为演绎推断概率因果主张,而应视为扩充性推断。本文最后将讨论证据等级制度,并为循证医学的立场辩护,即RCT是评估治疗效果的最佳可用方法。

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本文引用的文献

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Healthcare outcomes assessed with observational study designs compared with those assessed in randomized trials.与随机试验中评估的医疗保健结果相比,观察性研究设计评估的医疗保健结果。
Cochrane Database Syst Rev. 2014 Apr 29;2014(4):MR000034. doi: 10.1002/14651858.MR000034.pub2.
2
Why and for what are clinical trials the gold standard?为什么临床试验是金标准?其目的是什么?
Scand J Public Health. 2014 Mar;42(13 Suppl):41-8. doi: 10.1177/1403494813516712.
3
Until RCT proven? On the asymmetry of evidence requirements for risk assessment.
在有运动干预的随机对照试验中,APOE4基因型对生理和认知健康的影响:一项系统评价和荟萃分析。
Trials. 2025 Jan 20;26(1):20. doi: 10.1186/s13063-024-08696-4.
4
The Fragility of Statistically Significant Binary Outcomes for Treating Achilles Tendinopathy: A Systematic Review of Randomized Trials.治疗跟腱病时具有统计学意义的二元结局的脆弱性:随机试验的系统评价
Foot Ankle Orthop. 2024 Nov 20;9(4):24730114241300160. doi: 10.1177/24730114241300160. eCollection 2024 Oct.
5
Effectiveness of pharmacogenomics on the response and remission of treatment-resistant depression: a meta-analysis of randomised controlled trials.药物基因组学对难治性抑郁症反应和缓解的有效性:一项随机对照试验的荟萃分析。
Gen Psychiatr. 2023 Dec 26;36(6):e101050. doi: 10.1136/gpsych-2023-101050. eCollection 2023.
6
The cost-effectiveness of pegcetacoplan in complement treatment-naïve adults with paroxysmal nocturnal hemoglobinuria in the USA.佩格司他单抗治疗美国阵发性睡眠性血红蛋白尿症初治成人的成本效果分析。
J Comp Eff Res. 2023 Oct;12(10):e230055. doi: 10.57264/cer-2023-0055. Epub 2023 Sep 1.
7
What Research Evidence Is Valid for Psychotherapy Research?哪些研究证据对心理治疗研究有效?
Front Psychiatry. 2021 Jan 11;11:625380. doi: 10.3389/fpsyt.2020.625380. eCollection 2020.
8
A mixed methods case study investigating how randomised controlled trials (RCTs) are reported, understood and interpreted in practice.一项混合方法案例研究,旨在调查随机对照试验(RCT)在实践中是如何报告、理解和解释的。
BMC Med Res Methodol. 2020 May 12;20(1):112. doi: 10.1186/s12874-020-01009-8.
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The Expanding Role of Real-World Evidence Trials in Health Care Decision Making.真实世界证据试验在医疗保健决策中的作用不断扩大。
J Diabetes Sci Technol. 2020 Jan;14(1):174-179. doi: 10.1177/1932296819832653. Epub 2019 Mar 6.
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Acetyl-L-carnitine for patients with hepatic encephalopathy.乙酰左旋肉碱用于肝性脑病患者。
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直到 RCT 证明吗?关于风险评估证据要求的不对称性。
J Eval Clin Pract. 2013 Jun;19(3):454-62. doi: 10.1111/jep.12039.
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What does randomisation achieve?随机化能实现什么?
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5
Against homeopathy--a utilitarian perspective.反对顺势疗法——一种功利主义的视角。
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The limitations of randomized controlled trials in predicting effectiveness.随机对照试验在预测疗效方面的局限性。
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Perils of evidence-based medicine.循证医学的风险
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