Clinical pharmacology as a cornerstone of orphan drug development

Edward D Bashaw; Shiew-Mei Huang; Timothy R Coté; Anne R Pariser; Christine E Garnett; Gilbert Burckart; Lei Zhang; Angela Y Men; Christine D Le; Rosane Charlab; Jogaro V Gobburu; Lawrence J Lesko

doi:10.1038/nrd3595

Clinical pharmacology as a cornerstone of orphan drug development

Nat Rev Drug Discov. 2011 Oct 31;10(11):795-6. doi: 10.1038/nrd3595.

Authors

Edward D Bashaw¹, Shiew-Mei Huang, Timothy R Coté, Anne R Pariser, Christine E Garnett, Gilbert Burckart, Lei Zhang, Angela Y Men, Christine D Le, Rosane Charlab, Jogaro V Gobburu, Lawrence J Lesko

Affiliation

¹ Office of Translational Sciences, Office of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland 20993, USA. [email protected]

PMID: 22037026
DOI: 10.1038/nrd3595

Abstract

A recent US Food and Drug Administration (FDA) advisory committee meeting highlighted the potential of clinical pharmacology to overcome challenges in orphan drug development.

MeSH terms

Animals
Drug Discovery / legislation & jurisprudence
Drug Discovery / trends*
Humans
Orphan Drug Production* / legislation & jurisprudence
Pharmacology, Clinical / legislation & jurisprudence
Pharmacology, Clinical / trends*
Rare Diseases / drug therapy
United States
United States Food and Drug Administration / legislation & jurisprudence
United States Food and Drug Administration / trends