Clinical development of gene therapy needs a tailored approach: A regulatory perspective from the European Union

Human Gene Therapy Clinical Development

Volumn 25, Issue 1, 2014, Pages 1-6

  Narayanan, Gopalan ^a   Cossu, Giulio ^b,c   Galli, Maria Cristina ^b,d   Flory, Egbert ^b,e   Ovelgonne, Hans ^b,f   Salmikangas, Paula ^b,g   Schneider, Christian K ^b,h,i   Trouvin, Jean Hugues ^b,j  

^a Biologicals Advice Limited   (United Kingdom)

^b EUROPEAN MEDICINES AGENCY   (United Kingdom)

^c UNIVERSITY OF MANCHESTER   (United Kingdom)

^d ISTITUTO SUPERIORE DI SANITÀ   (Italy)

^e PAUL EHRLICH INSTITUT   (Germany)

^f MEDICINES EVALUATION BOARD   (Netherlands)

^g Finnish Medicines Agency   (Finland)

^h Danish Health and Medicines Authority   (Denmark)

ⁱ INSTITUTE OF INFECTION IMMUNOLOGY   (Germany)

^j UNIVERSITÉ PARIS DESCARTES   (France)

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Author keywords

[No Author keywords available]

Indexed keywords

GENE THERAPY AGENT;

DRUG EFFICACY; DRUG SAFETY; EUROPEAN UNION; FOLLOW UP; GENE THERAPY; GENE VECTOR; HUMAN; IMMUNOGENICITY; PRIORITY JOURNAL; REVIEW; RISK MANAGEMENT;

CONSUMER PRODUCT SAFETY; EUROPEAN UNION; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MARKETING OF HEALTH SERVICES;

EID: 84904411409     PISSN: 23248637     EISSN: 23248645     Source Type: Journal    
DOI: 10.1089/humc.2013.230     Document Type: Review

References (12)

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3. English, V. (2011). Commentary: Glybera-when the experts disagree. MedNous. Available at http://www.mednous.com/ news/commentary-glybera-when- experts-disagree (accessed 9 Nov 2011).
4. European Medicines Agency. (2009). Concept paper on the revision of the note for guidance on the quality, pre-clinical and clinical aspects of gene transfer medicinal products. EMA/CHMP/GTWP/BWP/234523/2009.
5. European Medicines Agency. (2011). Guideline on the riskbased approach according to annex I, part IV of Directive 2001/83/EC applied to advanced therapy medicinal products: EMA/CAT/CPWP/686637/2011.
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9. Melchiorri, D., et al. (2013). Regulatory evaluation of Glybera in Europe-two committees, one mission. Nat. Rev. Drug. Discov. 12, 719.
10. Miller, N. (2012). Glybera and the future of gene therapy in the European Union. Nat. Rev. Drug Discov. 11, 419. Reflection paper on design modifications of gene therapy medicinal products during development EMA/CAT/GTWP/ 44236/2009
11. Regulation (EC) No. 1394/2007 of the European Parliament and of the Council on Advanced Therapy Medicinal Products and amending Directive 2001/83/EC and Regulation (EC) No. 726/2004 and Directive 2009/120/EC amending Directive 2001/83/EC.
12. Vestergaard, H.T., DApote, L., Schneider, C.K., and Herberts, C. (2013). The evolution of nonclinical regulatory science: advanced therapy medicinal products as a paradigm. Mol. Ther. 21, 1644-1648.