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dc.contributor.authorO’Connor, Daniel J.
dc.contributor.authorBuckland, Jenny
dc.contributor.authorAlmond, Neil
dc.contributor.authorBoyle, Jennifer
dc.contributor.authorCoxon, Carmen
dc.contributor.authorGaki, Eleni
dc.contributor.authorMartin, Javier
dc.contributor.authorMattiuzzo, Giada
dc.contributor.authorMetcalfe, Clive
dc.contributor.authorPage, Mark
dc.contributor.authorRose, Nicola
dc.contributor.authorValdazo-Gonzalez, Begona
dc.contributor.authorZhao, Yuan
dc.contributor.authorSchneider, Christian K.
dc.creatorO’Connor, D.
dc.date.accessioned2019-09-18T09:42:40Z
dc.date.available2019-09-18T09:42:40Z
dc.date.issued2019-01-01
dc.identifier.doi10.1080/21678707.2019.1652598
dc.identifier.urihttps://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85070942310&origin=inward
dc.identifier.urihttp://hdl.handle.net/10033/621948
dc.description.abstractIntroduction: Standardization is important across the life cycle of medicinal products, supporting the diagnosis, treatment, and prevention of a wide range of diseases. For rare diseases, standardization is even more important, as patient groups are small, presenting significant challenges in the design, conduct, analysis, and interpretation of clinical studies. It is here that standardization institutions, including the UK’s National Institute for Biological Standards and Control (NIBSC), can have a key role. Areas covered: A considerable proportion of NIBSC’s work supports the better understanding, diagnosis, treatment, and prevention of rare diseases. NIBSC is also part of the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), creating an agency that is uniquely placed to combine scientific and regulatory expertize for the benefit of public health. This review provides an overview of NIBSC’s work in rare diseases and highlights the positive impact of the work of standardization institutions in this field. Expert opinion: Standardization in product development is key for patients with rare diseases. The work of standardization institutions is increasingly being recognized as crucial for supporting scientific and clinical advancements, and early and collaborative interactions can provide drug developers with the necessary expertize, when standards matter most.en_US
dc.language.isoenen_US
dc.publisherTaylor& Francisen_US
dc.relation.ispartofExpert Opinion on Orphan Drugs
dc.relation.ispartofseries7-8en_US
dc.rightsAttribution-NonCommercial-ShareAlike 4.0 International*
dc.rights.urihttp://creativecommons.org/licenses/by-nc-sa/4.0/*
dc.subjectdiagnosticsen_US
dc.subjectprophylaxisen_US
dc.subjectRare diseasesen_US
dc.subjectreference materialsen_US
dc.subjectstandardizationen_US
dc.subjectstandardization institutionsen_US
dc.subjectsurveillanceen_US
dc.subjecttherapeuticsen_US
dc.titleCommonly setting biological standards in rare diseasesen_US
dc.typeArticleen_US
dc.contributor.departmentTWINCORE, Zentrum für experimentelle und klinische Infektionsforschung GmbH,Feodor-Lynen Str. 7, 30625 Hannover, Germany.en_US
dc.identifier.journalExpert Opinion on Orphan Drugsen_US
dc.identifier.eid2-s2.0-85070942310
dc.identifier.scopusidSCOPUS_ID:85070942310
dc.relation.volume7
refterms.dateFOA2019-09-18T09:42:41Z


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