doi: 10.1038/clpt.2012.80.
Epub 2012 Jun 27.
Quantitative analysis to guide orphan drug development
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- PMID: 22739138
- DOI: 10.1038/clpt.2012.80
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Quantitative analysis to guide orphan drug development
Clin Pharmacol Ther.
2012 Aug.
Abstract
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.
Comment in
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An aide memoire for model-based drug development--a new key for the interpretation of the erythromycin breath test--therapeutic index as a biomarker for smart drug development.Biomark Med. 2013 Feb;7(1):89-91. doi: 10.2217/bmm.12.101. Biomark Med. 2013. PMID: 23387489 No abstract available.
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