title: Marketing Regulatory Oversight of Advanced Therapy Medicinal Products (ATMPs) in Europe: The EMA/CAT Perspective.
abstract: With the release of Regulation 1394/2007, a new framework for gene and cell therapy medicinal products and tissue-engineered products was established in the European Union. For all three product classes, called advanced therapy medicinal products, a centralised marketing authorisation became mandatory. The European Medicines Agency (EMA) together with its Committee for Advanced Therapies, Committee for Human Medicinal Products and the network of national agencies is responsible for scientific evaluation of the marketing authorisation applications. For a new application, data and information relating to manufacturing processes and quality control of the active substance and the final product have to be submitted for evaluation together with data from non-clinical and clinical safety and efficacy studies. Technical requirements for ATMPs are defined in the legislation, and guidance for different products is available through several EMA/CAT guidelines. Due to the diversity of ATMPs, a tailored approach for regulating these products is considered necessary. Thus, a risk-based approach has been introduced for ATMPs allowing flexibility for the regulatory requirements. Since the regulatory framework for ATMPs was established, five products have been licenced in the European Union. However, the pipeline of new ATMPs is much bigger, as seen from the significant numbers of different products discussed by the CAT in scientific advice and classification procedures. In 2013, a public consultation on the ATMP Regulation was conducted by the European Commission, and the results were published in 2014. The report proposes several improvements for the current framework and established procedures for the regulation of ATMPs.
authors.affiliation: Committee for Advanced Therapies (CAT), CAT Chairperson, European Medicines Agency, London, UK. Paula.Salmikangas@fimea.fi.|Department of Marketing Authorizations, Finnish Medicines Agency, Mannerheimintie 103b, 55, Helsinki, 00034, Finland. Paula.Salmikangas@fimea.fi.
authors.forename: Paula
authors.initials: P
authors.lastname: Salmikangas
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency, London, UK. martina.schuessler-lenz@pei.de.|Division of Medical Biotechnology, Paul-Ehrlich-Institut-Federal Institute for Vaccines and Biomedicines, Langen, 63225, Germany. martina.schuessler-lenz@pei.de.
authors.forename: Martina
authors.initials: M
authors.lastname: Schuessler-Lenz
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency, London, UK. sruiz@aemps.es.|Biologics and Biotechnology, Spanish Agency of Medicines and Medical Devices, Madrid, Spain. sruiz@aemps.es.
authors.forename: Sol
authors.initials: S
authors.lastname: Ruiz
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency, London, UK. Patrick.celis@ema.europa.eu.
authors.forename: Patrick
authors.initials: P
authors.lastname: Celis
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency, London, UK. ilona.reischl@ages.at.|Austrian Federal Office for Safety in Health Care/Austrian Medicines and Medical Devices Agency (BASG/AGES), Vienna, Austria. ilona.reischl@ages.at.
authors.forename: Ilona
authors.initials: I
authors.lastname: Reischl
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency, London, UK. margarida.menezes@infarmed.pt.|National Authority of Medicines and Health Products, Parque de Saúde de Lisboa, Avenida do Brasil 53, Lisbon, 1749-004, Portugal. margarida.menezes@infarmed.pt.
authors.forename: Margarida
authors.initials: M
authors.lastname: Menezes-Ferreira
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency, London, UK. floeg@pei.de.|Division of Medical Biotechnology, Paul-Ehrlich-Institut-Federal Institute for Vaccines and Biomedicines, Langen, 63225, Germany. floeg@pei.de.
authors.forename: Egbert
authors.initials: E
authors.lastname: Flory
authors.affiliation: Division of Medical Biotechnology, Paul-Ehrlich-Institute-Federal Institute for Vaccines and Biomedicines, Paul-Ehrlich-Straße 51-59, Langen, 63225, Germany. matthias.Renner@pei.de.
authors.forename: Matthias
authors.initials: M
authors.lastname: Renner
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency, London, UK. Nicolas.ferry@ansm.sante.fr.|BIOVAC Directorate, National Safety Agency for Drugs and Health Products, Management of Organic products, Saint Denis, France. Nicolas.ferry@ansm.sante.fr.
authors.forename: Nicolas
authors.initials: N
authors.lastname: Ferry
mesh_terms: D000818:Animals; D064987:Cell- and Tissue-Based Therapy / Q000941:ethics* / Q000379:methods; D002986:Clinical Trials as Topic; D004353:Drug Evaluation, Preclinical; D004335:Drug and Narcotic Control / Q000331:legislation & jurisprudence*; D015507:Drugs, Investigational / Q000493:pharmacokinetics / Q000494:pharmacology; D005060:Europe; D015316:Genetic Therapy / Q000941:ethics / Q000331:legislation & jurisprudence*; D006801:Humans; D017387:Investigational New Drug Application / Q000331:legislation & jurisprudence; D040541:Marketing / Q000331:legislation & jurisprudence*; D061214:Patient Safety / Q000331:legislation & jurisprudence; D017410:Practice Guidelines as Topic; D011786:Quality Control; D012107:Research Design; D057170:Translational Research, Biomedical / Q000941:ethics / Q000331:legislation & jurisprudence*
chemical_list: D015507:Drugs, Investigational
keywords: ATMP; Cell therapy; Gene therapy; Regulation; Risk-based approach; Tissue engineering