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Challenges with advanced therapy medicinal products and how to meet them

Abstract

Advanced therapy medicinal products (ATMPs), which include gene therapy medicinal products, somatic cell therapy medicinal products and tissue-engineered products, are at the cutting edge of innovation and offer a major hope for various diseases for which there are limited or no therapeutic options. They have therefore been subject to considerable interest and debate. Following the European regulation on ATMPs, a consolidated regulatory framework for these innovative medicines has recently been established. Central to this framework is the Committee for Advanced Therapies (CAT) at the European Medicines Agency (EMA), comprising a multidisciplinary scientific expert committee, representing all EU member states and European Free Trade Association countries, as well as patient and medical associations. In this article, the CAT discusses some of the typical issues raised by developers of ATMPs, and highlights the opportunities for such companies and research groups to approach the EMA and the CAT as a regulatory advisor during development.

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Figure 1: Expertise in the field of advanced therapy medicinal products declared by members and alternates of the Committee for Advanced Therapies.

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Acknowledgements

The CAT Chairman wishes to thank those CAT colleagues who took as 'Rapporteurs' the lead in the discussions of the subsections of this article to coordinate the participation of the entire Committee, namely: P. Salmikangas (CAT Vice-Chair and Finnish CAT member) and A. Tsiftsoglou (Greek CAT member) for CBMPs; T. VandenDriessche (Alternate for Clinicians' Representative) for the gene therapy section; W. Cheng (Swedish CAT Alternate) for the section on combined ATMPs; and P. Celis (EMA CAT Scientific Secretariat) for the section on how the CAT is involved in the development of ATMPs.

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For details of competing financial interests for each member please see the Committee for Advanced Therapeutics website (http://www.ema.europa.eu/htms/general/contacts/CAT/CAT_members.html).

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FURTHER INFORMATION

EMEA Innovation Task Force

EMEA SME Office

EMEA Scientific Guidelines for Biologicals, including guidelines specific to ATMPs:

EMEA Multidisciplinary Guidelines, including guidelines specific to ATMPs

Information on advanced therapies from the EMA

Information on CAT Members

Information on Medicines and Emerging Science

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The Committee for Advanced Therapies (CAT). Challenges with advanced therapy medicinal products and how to meet them. Nat Rev Drug Discov 9, 195–201 (2010). https://doi.org/10.1038/nrd3052

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