title: 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.
authors.forename: Christian K
authors.initials: CK
authors.lastname: Schneider
authors.forename: Paula
authors.initials: P
authors.lastname: Salmikangas
authors.forename: Bernd
authors.initials: B
authors.lastname: Jilma
authors.forename: Bruno
authors.initials: B
authors.lastname: Flamion
authors.forename: Lyubina Racheva
authors.initials: LR
authors.lastname: Todorova
authors.forename: Anna
authors.initials: A
authors.lastname: Paphitou
authors.forename: Ivana
authors.initials: I
authors.lastname: Haunerova
authors.forename: Toivo
authors.initials: T
authors.lastname: Maimets
authors.forename: Jean-Hugues
authors.initials: JH
authors.lastname: Trouvin
authors.forename: Egbert
authors.initials: E
authors.lastname: Flory
authors.forename: Asterios
authors.initials: A
authors.lastname: Tsiftsoglou
authors.forename: Balázs
authors.initials: B
authors.lastname: Sarkadi
authors.forename: Kolbeinn
authors.initials: K
authors.lastname: Gudmundsson
authors.forename: Maura
authors.initials: M
authors.lastname: O'Donovan
authors.forename: Giovanni
authors.initials: G
authors.lastname: Migliaccio
authors.forename: Jānis
authors.initials: J
authors.lastname: Ancāns
authors.forename: Romaldas
authors.initials: R
authors.lastname: Maciulaitis
authors.forename: Jean-Louis
authors.initials: JL
authors.lastname: Robert
authors.forename: Anthony
authors.initials: A
authors.lastname: Samuel
authors.forename: Johannes H
authors.initials: JH
authors.lastname: Ovelgönne
authors.forename: Marit
authors.initials: M
authors.lastname: Hystad
authors.forename: Andrzej Mariusz
authors.initials: AM
authors.lastname: Fal
authors.forename: Beatriz Silva
authors.initials: BS
authors.lastname: Lima
authors.forename: Anca Stela
authors.initials: AS
authors.lastname: Moraru
authors.forename: Peter
authors.initials: P
authors.lastname: Turcáni
authors.forename: Robert
authors.initials: R
authors.lastname: Zorec
authors.forename: Sol
authors.initials: S
authors.lastname: Ruiz
authors.forename: Lennart
authors.initials: L
authors.lastname: Akerblom
authors.forename: Gopalan
authors.initials: G
authors.lastname: Narayanan
authors.forename: Alastair
authors.initials: A
authors.lastname: Kent
authors.forename: Fabrizia
authors.initials: F
authors.lastname: Bignami
authors.forename: J George
authors.initials: JG
authors.lastname: Dickson
authors.forename: Dietger
authors.initials: D
authors.lastname: Niederwieser
authors.forename: María-Angeles
authors.initials: MA
authors.lastname: Figuerola-Santos
authors.forename: Ilona G
authors.initials: IG
authors.lastname: Reischl
authors.forename: Claire
authors.initials: C
authors.lastname: Beuneu
authors.forename: Rosen
authors.initials: R
authors.lastname: Georgiev
authors.forename: Maria
authors.initials: M
authors.lastname: Vassiliou
authors.forename: Alena
authors.initials: A
authors.lastname: Pychova
authors.forename: Mette
authors.initials: M
authors.lastname: Clausen
authors.forename: Taina
authors.initials: T
authors.lastname: Methuen
authors.forename: Sophie
authors.initials: S
authors.lastname: Lucas
authors.forename: Martina
authors.initials: M
authors.lastname: Schüssler-Lenz
authors.forename: Vasilios
authors.initials: V
authors.lastname: Kokkas
authors.forename: Zsuzsanna
authors.initials: Z
authors.lastname: Buzás
authors.forename: Niall
authors.initials: N
authors.lastname: MacAleenan
authors.forename: Maria Cristina
authors.initials: MC
authors.lastname: Galli
authors.forename: Aija
authors.initials: A
authors.lastname: Linē
authors.forename: Jolanta
authors.initials: J
authors.lastname: Gulbinovic
authors.forename: Guy
authors.initials: G
authors.lastname: Berchem
authors.forename: Mariusz
authors.initials: M
authors.lastname: Fraczek
authors.forename: Margarida
authors.initials: M
authors.lastname: Menezes-Ferreira
authors.forename: Nela
authors.initials: N
authors.lastname: Vilceanu
authors.forename: Mikulás
authors.initials: M
authors.lastname: Hrubisko
authors.forename: Petra
authors.initials: P
authors.lastname: Marinko
authors.forename: Marcos
authors.initials: M
authors.lastname: Timón
authors.forename: Wing
authors.initials: W
authors.lastname: Cheng
authors.forename: George Andrew
authors.initials: GA
authors.lastname: Crosbie
authors.forename: Nick
authors.initials: N
authors.lastname: Meade
authors.forename: Michelino Lipucci
authors.initials: ML
authors.lastname: di Paola
authors.forename: Thierry
authors.initials: T
authors.lastname: VandenDriessche
authors.forename: Per
authors.initials: P
authors.lastname: Ljungman
authors.forename: Lucia
authors.initials: L
authors.lastname: D'Apote
authors.forename: Olga
authors.initials: O
authors.lastname: Oliver-Diaz
authors.forename: Isabel
authors.initials: I
authors.lastname: Büttel
authors.forename: Patrick
authors.initials: P
authors.lastname: Celis
mesh_terms: D005062:European Union; D015316:Genetic Therapy / Q000331:legislation & jurisprudence* / Q000379:methods; D033161:Government Regulation; D006801:Humans; D033581:Stem Cell Transplantation / Q000331:legislation & jurisprudence* / Q000379:methods; D023822:Tissue Engineering / Q000331:legislation & jurisprudence*