代表性与外推:来自临床试验的证据

Representation and Extrapolation: Evidence from Clinical Trials

Quarterly Journal of Economics · 2023
被引 21
人大 A+FT50ABS 4*

中文导读

研究黑人患者在临床试验中代表性不足的原因和后果,发现代表性样本能提高医生处方意愿和患者对药效的信任,但过去受益多的群体当前更易招募,导致代表性持续不足。

Abstract

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.

临床试验代表性外推患者入组