Quantitative Analysis to Guide Orphan Drug Development

被引：6
|
作者：
Lesko, L. J. [1 ]
机构：
[1] Univ Florida, Dept Pharmaceut, Ctr Pharmacometr & Syst Pharmacol, Orlando, FL USA
来源：
CLINICAL PHARMACOLOGY & THERAPEUTICS | 2012年 / 92卷 / 02期
关键词：
RARE DISEASES; CHILDREN;
D O I：
10.1038/clpt.2012.80
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
The development of orphan drugs for rare diseases has made impressive strides in the past 10 years. There has been a surge in orphan drug designations, but new drug approvals have not kept up. This article presents a three-pronged hierarchical strategy for quantitative analysis of data at the descriptive, mechanistic, and systems levels of the biological system that could represent a standardized and rational approach to orphan drug development. Examples are provided to illustrate the concept.
引用
收藏
页码：258 / 261
页数：4
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