无知是福:药物审批中的故意不可知论

When ignorance is bliss: Intentional agnosticism in drug approval

Health Economics · 2019
被引 1
人大 A-

中文导读

研究了公共卫生机构在药物审批中为何故意忽略部分人群的治疗效果,即使有机会改变也不要求更包容的试验,并提出了一个机制解释这种故意不可知论。

Abstract

In developed nations, public health agencies typically use data from randomized controlled trials to evaluate new drugs. However, these trials routinely exclude populations to which clinicians prescribe approved drugs, meaning some patients are treated with drugs, which were approved on the basis of another group's treatment response. Despite having opportunities to change, some health agencies have not mandated greater inclusion in drug trials and appear to prefer remaining ignorant of some populations' treatment effects when approving a drug. To explore this decision, I introduce a novel mechanism by which a health agency would choose to be intentionally agnostic regarding a population's treatment response. The main contribution of this paper is in showing how ambiguity about on-label and off-label prescription rates could possibly encourage population exclusion in drug approval trials even in the absence of concerns about trial necessity or cost.

故意不可知论药物审批人群排除标签外处方