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Marketing Regulatory Oversight of Advanced Therapy Medicinal Products (ATMPs) in Europe: The EMA/CAT Perspective.
Adv Exp Med Biol. 2015; 871:103-30.AE

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+Show Affiliations

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.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.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.Committee for Advanced Therapies (CAT), European Medicines Agency, London, UK. Patrick.celis@ema.europa.eu.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.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.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.Division of Medical Biotechnology, Paul-Ehrlich-Institute-Federal Institute for Vaccines and Biomedicines, Paul-Ehrlich-Straβe 51-59, Langen, 63225, Germany. matthias.Renner@pei.de.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.

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

26374215

Citation

Salmikangas, Paula, et al. "Marketing Regulatory Oversight of Advanced Therapy Medicinal Products (ATMPs) in Europe: the EMA/CAT Perspective." Advances in Experimental Medicine and Biology, vol. 871, 2015, pp. 103-30.
Salmikangas P, Schuessler-Lenz M, Ruiz S, et al. Marketing Regulatory Oversight of Advanced Therapy Medicinal Products (ATMPs) in Europe: The EMA/CAT Perspective. Adv Exp Med Biol. 2015;871:103-30.
Salmikangas, P., Schuessler-Lenz, M., Ruiz, S., Celis, P., Reischl, I., Menezes-Ferreira, M., Flory, E., Renner, M., & Ferry, N. (2015). Marketing Regulatory Oversight of Advanced Therapy Medicinal Products (ATMPs) in Europe: The EMA/CAT Perspective. Advances in Experimental Medicine and Biology, 871, 103-30. https://doi.org/10.1007/978-3-319-18618-4_6
Salmikangas P, et al. Marketing Regulatory Oversight of Advanced Therapy Medicinal Products (ATMPs) in Europe: the EMA/CAT Perspective. Adv Exp Med Biol. 2015;871:103-30. PubMed PMID: 26374215.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Marketing Regulatory Oversight of Advanced Therapy Medicinal Products (ATMPs) in Europe: The EMA/CAT Perspective. AU - Salmikangas,Paula, AU - Schuessler-Lenz,Martina, AU - Ruiz,Sol, AU - Celis,Patrick, AU - Reischl,Ilona, AU - Menezes-Ferreira,Margarida, AU - Flory,Egbert, AU - Renner,Matthias, AU - Ferry,Nicolas, PY - 2015/9/17/entrez PY - 2015/9/17/pubmed PY - 2015/12/30/medline KW - ATMP KW - Cell therapy KW - Gene therapy KW - Regulation KW - Risk-based approach KW - Tissue engineering SP - 103 EP - 30 JF - Advances in experimental medicine and biology JO - Adv. Exp. Med. Biol. VL - 871 N2 - 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. SN - 0065-2598 UR - https://www.unboundmedicine.com/medline/citation/26374215/Marketing_Regulatory_Oversight_of_Advanced_Therapy_Medicinal_Products__ATMPs__in_Europe:_The_EMA/CAT_Perspective_ L2 - https://dx.doi.org/10.1007/978-3-319-18618-4_6 DB - PRIME DP - Unbound Medicine ER -