Clinical pharmacology as a cornerstone of orphan drug development.
A recent US Food and Drug Administration (FDA) advisory committee meeting highlighted the potential of clinical pharmacology to overcome challenges in orphan drug development.
Office of Translational Sciences, Office of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland 20993, USA. Edward.bashaw@fda.hhs.gov
Edward D
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Bashaw
Shiew-Mei
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Huang
Timothy R
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Coté
Anne R
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Pariser
Christine E
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Garnett
Gilbert
G
Burckart
Lei
L
Zhang
Angela Y
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Men
Christine D
CD
Le
Rosane
R
Charlab
Jogaro V
JV
Gobburu
Lawrence J
LJ
Lesko
D000818:Animals; D055808:Drug Discovery / Q000331:legislation & jurisprudence / Q000639:trends*; D006801:Humans; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence; D010601:Pharmacology, Clinical / Q000331:legislation & jurisprudence / Q000639:trends*; D035583:Rare Diseases / Q000188:drug therapy; D014481:United States; D014486:United States Food and Drug Administration / Q000331:legislation & jurisprudence / Q000639:trends