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...

Full description

Saved in:
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
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
cited_by cdi_FETCH-LOGICAL-c3557-70550e5d8c930845490194312763f0d57d9774e006caa313b1f9b193d36f77a83
cites cdi_FETCH-LOGICAL-c3557-70550e5d8c930845490194312763f0d57d9774e006caa313b1f9b193d36f77a83
container_end_page 329
container_issue 4
container_start_page 327
container_title Clinical pharmacology and therapeutics
container_volume 100
creator Bashaw, ED
description 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.
doi_str_mv 10.1002/cpt.427
format article
fullrecord <record><control><sourceid>proquest_cross</sourceid><recordid>TN_cdi_proquest_miscellaneous_1818340555</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>1818340555</sourcerecordid><originalsourceid>FETCH-LOGICAL-c3557-70550e5d8c930845490194312763f0d57d9774e006caa313b1f9b193d36f77a83</originalsourceid><addsrcrecordid>eNp1kNFKwzAUhoMoOqf4BpI7BakmTdM0l3PoVMb0YiJehSw9ndVuqUk73dsb6dydV4cD3_nPz4fQCSWXlJD4ytTNZRKLHdSjnMVRyhnfRT1CiIxkzNIDdOj9e1gTmWX76CAWCRU8kT30OsCmKpel0RWu37RbaGMrO19HDuZtpRvr1rgG52swTbkCbJe4eQOs69rZVbixBc5dO_e4sA477QDnpQftwR-hvUJXHo43s4-eb2-mw7to_Di6Hw7GkWGci0gQzgnwPDOSkSwJpQiVCaOxSFlBci5yKUQChKRGa0bZjBZyRiXLWVoIoTPWR-ddbmj02YJv1KL0BqpKL8G2XtGMZiwJX3hAzzrUOOu9g0LVrlxot1aUqF-PKnhUwWMgTzeh7WwB-Zb7ExeAiw74KitY_5ejhk_TLi7q6NI38L2ltftQqWCCq5fJSEkx4TS7HqkH9gP9cYmi</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>1818340555</pqid></control><display><type>article</type><title>A clinical pharmacology-regulatory perspective on the approval of drugs for rare diseases</title><source>Wiley</source><creator>Bashaw, ED</creator><creatorcontrib>Bashaw, ED</creatorcontrib><description>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.</description><identifier>ISSN: 0009-9236</identifier><identifier>EISSN: 1532-6535</identifier><identifier>DOI: 10.1002/cpt.427</identifier><identifier>PMID: 27417549</identifier><language>eng</language><publisher>United States: Blackwell Publishing Ltd</publisher><subject>Drug Approval ; Drug Discovery - standards ; Humans ; Orphan Drug Production - standards ; Rare Diseases - drug therapy ; United States ; United States Food and Drug Administration</subject><ispartof>Clinical pharmacology and therapeutics, 2016-10, Vol.100 (4), p.327-329</ispartof><rights>Published 2016. This article is a U.S. Government work and is in the public domain in the USA.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c3557-70550e5d8c930845490194312763f0d57d9774e006caa313b1f9b193d36f77a83</citedby><cites>FETCH-LOGICAL-c3557-70550e5d8c930845490194312763f0d57d9774e006caa313b1f9b193d36f77a83</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,780,784,27924,27925</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/27417549$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Bashaw, ED</creatorcontrib><title>A clinical pharmacology-regulatory perspective on the approval of drugs for rare diseases</title><title>Clinical pharmacology and therapeutics</title><addtitle>Clin. Pharmacol. Ther</addtitle><description>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.</description><subject>Drug Approval</subject><subject>Drug Discovery - standards</subject><subject>Humans</subject><subject>Orphan Drug Production - standards</subject><subject>Rare Diseases - drug therapy</subject><subject>United States</subject><subject>United States Food and Drug Administration</subject><issn>0009-9236</issn><issn>1532-6535</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2016</creationdate><recordtype>article</recordtype><recordid>eNp1kNFKwzAUhoMoOqf4BpI7BakmTdM0l3PoVMb0YiJehSw9ndVuqUk73dsb6dydV4cD3_nPz4fQCSWXlJD4ytTNZRKLHdSjnMVRyhnfRT1CiIxkzNIDdOj9e1gTmWX76CAWCRU8kT30OsCmKpel0RWu37RbaGMrO19HDuZtpRvr1rgG52swTbkCbJe4eQOs69rZVbixBc5dO_e4sA477QDnpQftwR-hvUJXHo43s4-eb2-mw7to_Di6Hw7GkWGci0gQzgnwPDOSkSwJpQiVCaOxSFlBci5yKUQChKRGa0bZjBZyRiXLWVoIoTPWR-ddbmj02YJv1KL0BqpKL8G2XtGMZiwJX3hAzzrUOOu9g0LVrlxot1aUqF-PKnhUwWMgTzeh7WwB-Zb7ExeAiw74KitY_5ejhk_TLi7q6NI38L2ltftQqWCCq5fJSEkx4TS7HqkH9gP9cYmi</recordid><startdate>201610</startdate><enddate>201610</enddate><creator>Bashaw, ED</creator><general>Blackwell Publishing Ltd</general><scope>BSCLL</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope></search><sort><creationdate>201610</creationdate><title>A clinical pharmacology-regulatory perspective on the approval of drugs for rare diseases</title><author>Bashaw, ED</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c3557-70550e5d8c930845490194312763f0d57d9774e006caa313b1f9b193d36f77a83</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2016</creationdate><topic>Drug Approval</topic><topic>Drug Discovery - standards</topic><topic>Humans</topic><topic>Orphan Drug Production - standards</topic><topic>Rare Diseases - drug therapy</topic><topic>United States</topic><topic>United States Food and Drug Administration</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Bashaw, ED</creatorcontrib><collection>Istex</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><jtitle>Clinical pharmacology and therapeutics</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Bashaw, ED</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>A clinical pharmacology-regulatory perspective on the approval of drugs for rare diseases</atitle><jtitle>Clinical pharmacology and therapeutics</jtitle><addtitle>Clin. Pharmacol. Ther</addtitle><date>2016-10</date><risdate>2016</risdate><volume>100</volume><issue>4</issue><spage>327</spage><epage>329</epage><pages>327-329</pages><issn>0009-9236</issn><eissn>1532-6535</eissn><abstract>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.</abstract><cop>United States</cop><pub>Blackwell Publishing Ltd</pub><pmid>27417549</pmid><doi>10.1002/cpt.427</doi><tpages>3</tpages></addata></record>
fulltext fulltext
identifier ISSN: 0009-9236
ispartof Clinical pharmacology and therapeutics, 2016-10, Vol.100 (4), p.327-329
issn 0009-9236
1532-6535
language eng
recordid cdi_proquest_miscellaneous_1818340555
source Wiley
subjects Drug Approval
Drug Discovery - standards
Humans
Orphan Drug Production - standards
Rare Diseases - drug therapy
United States
United States Food and Drug Administration
title A clinical pharmacology-regulatory perspective on the approval of drugs for rare diseases
url http://sfxeu10.hosted.exlibrisgroup.com/loughborough?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-01-07T17%3A43%3A48IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_cross&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=A%20clinical%20pharmacology-regulatory%20perspective%20on%20the%20approval%20of%20drugs%20for%20rare%20diseases&rft.jtitle=Clinical%20pharmacology%20and%20therapeutics&rft.au=Bashaw,%20ED&rft.date=2016-10&rft.volume=100&rft.issue=4&rft.spage=327&rft.epage=329&rft.pages=327-329&rft.issn=0009-9236&rft.eissn=1532-6535&rft_id=info:doi/10.1002/cpt.427&rft_dat=%3Cproquest_cross%3E1818340555%3C/proquest_cross%3E%3Cgrp_id%3Ecdi_FETCH-LOGICAL-c3557-70550e5d8c930845490194312763f0d57d9774e006caa313b1f9b193d36f77a83%3C/grp_id%3E%3Coa%3E%3C/oa%3E%3Curl%3E%3C/url%3E&rft_id=info:oai/&rft_pqid=1818340555&rft_id=info:pmid/27417549&rfr_iscdi=true