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Quantitative Analysis to Guide Orphan Drug Development
Author(s) -
Lesko L J
Publication year - 2012
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1038/clpt.2012.80
Subject(s) - orphan drug , clinical pharmacology , drug development , pharmacology , drug , medicine , bioinformatics , biology
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. Clinical Pharmacology & Therapeutics (2012); 92 2, 258–261. doi: 10.1038/clpt.2012.80