Quantitative analysis to guide orphan drug development.
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.
Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, University of Florida, Orlando, FL, USA. llesko@cop.ufl.edu
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Lesko
D002986:Clinical Trials as Topic; D017277:Drug Approval / Q000191:economics / Q000331:legislation & jurisprudence / Q000379:methods*; D055808:Drug Discovery / Q000191:economics / Q000331:legislation & jurisprudence / Q000379:methods*; D006801:Humans; D008954:Models, Biological; D009965:Orphan Drug Production / Q000191:economics / Q000331:legislation & jurisprudence / Q000379:methods*; D012107:Research Design / Q000331:legislation & jurisprudence / Q000592:standards*