Challenges with advanced therapy medicinal products and how to meet them

Christian K. Schneider, Patrick Celis, Paula Salmikangas, María Angeles Figuerola-Santos, Lucia D'Apote, Olga Oliver-Diaz, Isabel Büttel, Romaldas Mačiulaitis, Jean Louis Robert, Beatriz Silva Lima, Sol Ruiz, Bernd Jilma, Bruno Flamion, Lyubina Racheva Todorova, Anna Paphitou, Ivana Haunerova, Mette Clausen, Toivo Maimets, Jean Hugues Trouvin, Egbert FloryAsterios Tsiftsoglou, Balázs Sarkadi, Kolbeinn Gudmundsson, Maura O'Donovan, Giovanni Migliaccio, Janis Ancans, Anthony Samuel, Johannes H. Ovelgönne, Marit Hystad, Andrzej Mariusz Fal, Anca Stela Moraru, Peter Turčáni, Robert Zorec, Lennart Åkerblom, Gopalan Narayanan, Alastair Kent, Fabrizia Bignami, J. George Dickson, Dietger Niederwieser

Research output: Contribution to journalComment/debate

129 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)195-201
Number of pages7
JournalNature Reviews Drug Discovery
Volume9
Issue number3
DOIs
Publication statusPublished - Jan 1 2010

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery

Fingerprint Dive into the research topics of 'Challenges with advanced therapy medicinal products and how to meet them'. Together they form a unique fingerprint.

  • Cite this

    Schneider, C. K., Celis, P., Salmikangas, P., Figuerola-Santos, M. A., D'Apote, L., Oliver-Diaz, O., Büttel, I., Mačiulaitis, R., Robert, J. L., Silva Lima, B., Ruiz, S., Jilma, B., Flamion, B., Racheva Todorova, L., Paphitou, A., Haunerova, I., Clausen, M., Maimets, T., Trouvin, J. H., ... Niederwieser, D. (2010). Challenges with advanced therapy medicinal products and how to meet them. Nature Reviews Drug Discovery, 9(3), 195-201. https://doi.org/10.1038/nrd3052