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代表性与外推法:来自临床试验的证据。

REPRESENTATION AND EXTRAPOLATION: EVIDENCE FROM CLINICAL TRIALS.

作者信息

Alsan Marcella, Durvasula Maya, Gupta Harsh, Schwartzstein Joshua, Williams Heidi

机构信息

Harvard Kennedy School and National Bureau of Economic Research, United States.

Stanford University, United States.

出版信息

Q J Econ. 2024 Feb;139(1):575-635. doi: 10.1093/qje/qjad036. Epub 2023 Sep 5.

Abstract

This article examines the consequences and causes of low enrollment of Black patients in clinical trials. We develop a simple model of similarity-based extrapolation that predicts that evidence is more relevant for decision-making by physicians and patients when it is more representative of the group being treated. This generates the key result that the perceived benefit of a medicine for a group depends not only on the average benefit from a trial but also on the share of patients from that group who were enrolled in the trial. In survey experiments, we find that physicians who care for Black patients are more willing to prescribe drugs tested in representative samples, an effect substantial enough to close observed gaps in the prescribing rates of new medicines. Black patients update more on drug efficacy when the sample that the drug is tested on is more representative, reducing Black-white patient gaps in beliefs about whether the drug will work as described. Despite these benefits of representative data, our framework and evidence suggest that those who have benefited more from past medical breakthroughs are less costly to enroll in the present, leading to persistence in who is represented in the evidence base.

摘要

本文探讨了黑人患者参与临床试验人数较少的后果及原因。我们构建了一个基于相似性推断的简单模型,该模型预测,当证据更能代表接受治疗的群体时,它对医生和患者的决策就更具相关性。这产生了一个关键结果,即一种药物对某一群体的感知益处不仅取决于试验中的平均益处,还取决于该群体中参与试验的患者比例。在调查实验中,我们发现照顾黑人患者的医生更愿意开在具有代表性样本中测试过的药物,这种影响足够大,足以弥合新药处方率中观察到的差距。当药物测试样本更具代表性时,黑人患者对药物疗效的更新更多,减少了黑人和白人患者在药物是否会如描述般起作用的信念上的差距。尽管代表性数据有这些益处,但我们的框架和证据表明,那些从过去医学突破中获益更多的人在当前参与试验的成本更低,导致证据基础中所代表的人群持续存在差异。

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