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A clinical pharmacology‐regulatory perspective on the approval of drugs for rare diseases
Author(s) -
Bashaw ED
Publication year - 2016
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 - 10.1002/cpt.427
Subject(s) - orphan drug , drug development , drug approval , food and drug administration , medicine , clinical pharmacology , drug , regulatory science , pharmacology , intensive care medicine , perspective (graphical) , bioinformatics , biology , pathology , artificial intelligence , computer science
Orphan drugs or drugs for rare diseases represents a particular regulatory conundrum. There is a desperate need for effective therapies for these patients, who have been historically underserved by the drug development community. However, there is also a need to make sure these therapies are both safe and effective. In response, the US Food and Drug Administration (FDA) has evolved new approaches to facilitate drug development in this area.

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