A clinical pharmacology-regulatory perspective on the approval of drugs for rare diseases

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 sa...

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Bibliographic Details
Published in:Clinical pharmacology and therapeutics 2016-10, Vol.100 (4), p.327-329
Main Author: Bashaw, ED
Format: Article
Language:English
Subjects:
Drug Approval
Drug Discovery - standards
Humans
Orphan Drug Production - standards
Rare Diseases - drug therapy
United States
United States Food and Drug Administration
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Summary: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.
ISSN:0009-9236
1532-6535
DOI:10.1002/cpt.427