Biomarkers and Surrogate Markers: An FDA Perspective

被引：61

作者：

Katz R. ^{[1
]}

机构：

[1] Division of Neuropharmacological Drug Products, United States Food and Drug Administration, Rockville

来源：

NeuroRX | 2004年 / 1卷 / 2期

关键词：

accelerated approval; biomarker; FDA; Surrogate marker; validation;

D O I：

10.1602/neurorx.1.2.189

中图分类号：

学科分类号：

摘要：

Interest is increasing rapidly in the use of surrogate markers as primary measures of the effectiveness of investigational drugs in definitive drug trials. Many such surrogate markers have been proposed as potential candidates for use in definitive effectiveness trials of agents to treat neurologic or psychiatric disease, but as of this date, there are no such markers that have been adequately "validated," that is, shown to predict the effect of the treatment on the clinical outcome of interest. While the current law and regulations permit the United States Food and Drug Administration to base the approval of a drug product on a determination the effect of the drug on an unvalidated surrogate marker (that is, one for which it is not known that an effect on the surrogate actually predicts the desired clinical benefit), there are a number of difficulties in interpreting trials that use surrogate markers as primary measures of drug effect. In this article, the relevant regulatory context will be discussed, as well as the epistemological problems related to the interpretation of clinical trials in which unvalidated surrogate markers are used as primary outcomes. © 2004 The American Society for Experimental NeuroTherapeutics, Inc.

引用

页码：189 / 195

页数：6

共 14 条

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