title: Clinical development of gene therapy needs a tailored approach: a regulatory perspective from the European Union.
abstract: Gene therapy is a rapidly evolving field that needs an integrated approach, as acknowledged in the concept article on the revision of the guideline on gene transfer medicinal products. The first gene therapy application for marketing authorization was approved in the International Conference on Harmonisation (ICH) region in 2012, the product being Alipogene tiparvovec. The regulatory process for this product has been commented on extensively, highlighting the challenges posed by such a novel technology. Here, as current or previous members of the Committee for Advanced Therapies, we share our perspectives and views on gene therapy as a treatment modality based on current common understanding and regulatory experience of gene therapy products in the European Union to date. It is our view that a tailored approach is needed for a given gene therapy product in order to achieve successful marketing authorization.
authors.affiliation: 1 Biologicals Advice Limited, KT3 3AP Surrey, United Kingdom .
authors.forename: Gopalan
authors.initials: G
authors.lastname: Narayanan
authors.forename: Giulio
authors.initials: G
authors.lastname: Cossu
authors.forename: Maria Cristina
authors.initials: MC
authors.lastname: Galli
authors.forename: Egbert
authors.initials: E
authors.lastname: Flory
authors.forename: Hans
authors.initials: H
authors.lastname: Ovelgonne
authors.forename: Paula
authors.initials: P
authors.lastname: Salmikangas
authors.forename: Christian K
authors.initials: CK
authors.lastname: Schneider
authors.forename: Jean-Hugues
authors.initials: JH
authors.lastname: Trouvin
mesh_terms: D003257:Consumer Product Safety / Q000331:legislation & jurisprudence*; D005062:European Union; D015316:Genetic Therapy / Q000331:legislation & jurisprudence*; D005822:Genetic Vectors / Q000009:adverse effects / Q000627:therapeutic use; D006801:Humans; D008389:Marketing of Health Services / Q000331:legislation & jurisprudence*