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