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dc.contributor.authorPearson, ADJ
dc.contributor.authorPfister, SM
dc.contributor.authorBaruchel, A
dc.contributor.authorBourquin, J-P
dc.contributor.authorCasanova, M
dc.contributor.authorChesler, L
dc.contributor.authorDoz, F
dc.contributor.authorEggert, A
dc.contributor.authorGeoerger, B
dc.contributor.authorJones, DTW
dc.contributor.authorKearns, PR
dc.contributor.authorMolenaar, JJ
dc.contributor.authorMorland, B
dc.contributor.authorSchleiermacher, G
dc.contributor.authorSchulte, JH
dc.contributor.authorVormoor, J
dc.contributor.authorMarshall, LV
dc.contributor.authorZwaan, CM
dc.contributor.authorVassal, G
dc.contributor.authorExecutive and Biology Committees of the Innovative Therapies for Children with Cancer European Consortium
dc.date.accessioned2018-02-16T10:15:49Z
dc.date.issued2017-07
dc.identifier.citationThe Lancet. Oncology, 2017, 18 (7), pp. e394 - e404
dc.identifier.issn1470-2045
dc.identifier.urihttps://repository.icr.ac.uk/handle/internal/1176
dc.identifier.eissn1474-5488
dc.identifier.doi10.1016/s1470-2045(17)30442-4
dc.description.abstractNew drugs are crucially needed for children with cancer. The European Paediatric Regulation facilitates paediatric class waivers for drugs developed for diseases only occurring in adults. In this Review, we retrospectively searched oncology drugs that were class waivered between June, 2012, and June, 2015. 147 oncology class waivers were confirmed for 89 drugs. Mechanisms of action were then assessed as potential paediatric therapeutic targets by both a literature search and an expert review. 48 (54%) of the 89 class-waivered drugs had a mechanisms of action warranting paediatric development. Two (2%) class-waivered drugs were considered not relevant and 16 (18%) required further data. In light of these results, we propose five initiatives: an aggregated database of paediatric biological tumour drug targets; molecular profiling of all paediatric tumours at diagnosis and relapse; a joint academic-pharmaceutical industry preclinical platform to help analyse the activity of new drugs (Innovative Therapy for Children with Cancer Paediatric Preclinical Proof-of-Concept Platform); paediatric strategy forums; and the suppression of article 11b of the European Paediatric Regulation, which allows product-specific waivers on the grounds that the associated condition does not occur in children. These initiatives and a mechanism of action-based approach to drug development will accelerate the delivery of new therapeutic drugs for front-line therapy for those children who have unmet medical needs.
dc.formatPrint
dc.format.extente394 - e404
dc.languageeng
dc.language.isoeng
dc.subjectExecutive and Biology Committees of the Innovative Therapies for Children with Cancer European Consortium
dc.subjectHumans
dc.subjectNeoplasms
dc.subjectAntineoplastic Agents
dc.subjectBiological Products
dc.subjectLegislation, Drug
dc.subjectAdolescent
dc.subjectChild
dc.subjectChild, Preschool
dc.subjectInfant
dc.subjectInfant, Newborn
dc.subjectEurope
dc.subjectDrug Discovery
dc.subjectPrecision Medicine
dc.titleFrom class waivers to precision medicine in paediatric oncology.
dc.typeJournal Article
dcterms.dateAccepted2017-05-04
rioxxterms.versionofrecord10.1016/s1470-2045(17)30442-4
rioxxterms.licenseref.startdate2017-07
rioxxterms.typeJournal Article/Review
dc.relation.isPartOfThe Lancet. Oncology
pubs.issue7
pubs.notesNot known
pubs.organisational-group/ICR
pubs.organisational-group/ICR/Primary Group
pubs.organisational-group/ICR/Primary Group/ICR Divisions
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Cancer Therapeutics
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Cancer Therapeutics/Paediatric Solid Tumour Biology and Therapeutics
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Clinical Studies
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Clinical Studies/Paediatric Solid Tumour Biology and Therapeutics
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Molecular Pathology
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Molecular Pathology/Paediatric Solid Tumour Biology and Therapeutics
pubs.organisational-group/ICR/Primary Group/Royal Marsden Clinical Units
pubs.organisational-group/ICR
pubs.organisational-group/ICR/Primary Group
pubs.organisational-group/ICR/Primary Group/ICR Divisions
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Cancer Therapeutics
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Cancer Therapeutics/Paediatric Solid Tumour Biology and Therapeutics
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Clinical Studies
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Clinical Studies/Paediatric Solid Tumour Biology and Therapeutics
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Molecular Pathology
pubs.organisational-group/ICR/Primary Group/ICR Divisions/Molecular Pathology/Paediatric Solid Tumour Biology and Therapeutics
pubs.organisational-group/ICR/Primary Group/Royal Marsden Clinical Units
pubs.publication-statusPublished
pubs.volume18
pubs.embargo.termsNot known
icr.researchteamPaediatric Solid Tumour Biology and Therapeuticsen_US
dc.contributor.icrauthorChesler, Louisen
dc.contributor.icrauthorMarsden,en


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