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Regulation of advanced therapy medicinal products in Europe and the role of academia

Pearce, Kim F.; Hildebrandt, Martin; Greinix, Hildegard; Scheding, Stefan LU ; Koehl, Ulrike; Worel, Nina; Apperley, Jane; Edinger, Matthius; Hauser, Andrea and Mischak-Weissinger, Eva, et al. (2014) In Cytotherapy 16(3). p.289-297
Abstract
Background aims. Advanced therapy medicinal products (ATMP) are gene therapy, somatic cell therapy or tissue-engineered products regulated under (EC) No. 1394/2007 to ensure their free movement within the European Union while guaranteeing the highest level of health protection for patients. Academic good manufacturing practice (GMP) centers are major contributors in the development of ATMPs and this study assessed the impact of regulations on them. Methods. European academic and non-industrial facilities (n = 747) were contacted, and a representative sample of 50 replied to a detailed questionnaire. Experienced centres were further selected in every Member State (MS) for semi-structured interviews. Indicators of ATMP production and... (More)
Background aims. Advanced therapy medicinal products (ATMP) are gene therapy, somatic cell therapy or tissue-engineered products regulated under (EC) No. 1394/2007 to ensure their free movement within the European Union while guaranteeing the highest level of health protection for patients. Academic good manufacturing practice (GMP) centers are major contributors in the development of ATMPs and this study assessed the impact of regulations on them. Methods. European academic and non-industrial facilities (n = 747) were contacted, and a representative sample of 50 replied to a detailed questionnaire. Experienced centres were further selected in every Member State (MS) for semi-structured interviews. Indicators of ATMP production and development success were statistically assessed, and opinions about directive implementation were documented. Results. Facilities experienced in manufacturing cell therapy transplant products are the most successful in developing ATMPs. New centres lacking this background struggle to enter the field, and there remains a shortage of facilities in academia participating in translational research. This is compounded by heterogeneous implementation of the regulations across MS. Conclusions. GMP facilities successfully developing ATMPs are present in all MS. However, the implementation of regulations is heterogeneous between MS, with substantial differences in the definition of ATMPs and in the approved manufacturing environment. The cost of GMP compliance is underestimated by research funding bodies. This is detrimental to development of new ATMPs and commercialization of any that are successful in early clinical trials. Academic GMP practitioners should strengthen their political visibility and contribute to the development of functional and effective European Union legislation in this field. (Less)
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publication status
published
subject
keywords
advanced therapy medicinal products, European Union, good manufacturing, practice, manufacturing, regulation
in
Cytotherapy
volume
16
issue
3
pages
289 - 297
publisher
Taylor & Francis
external identifiers
  • wos:000331593800002
  • scopus:84895170844
ISSN
1477-2566
DOI
10.1016/j.jcyt.2013.08.003
language
English
LU publication?
yes
id
90368b7e-71a4-4c9d-a2d4-50ee2e3d7575 (old id 4363662)
date added to LUP
2014-04-07 09:21:28
date last changed
2017-10-01 03:32:46
@article{90368b7e-71a4-4c9d-a2d4-50ee2e3d7575,
  abstract     = {Background aims. Advanced therapy medicinal products (ATMP) are gene therapy, somatic cell therapy or tissue-engineered products regulated under (EC) No. 1394/2007 to ensure their free movement within the European Union while guaranteeing the highest level of health protection for patients. Academic good manufacturing practice (GMP) centers are major contributors in the development of ATMPs and this study assessed the impact of regulations on them. Methods. European academic and non-industrial facilities (n = 747) were contacted, and a representative sample of 50 replied to a detailed questionnaire. Experienced centres were further selected in every Member State (MS) for semi-structured interviews. Indicators of ATMP production and development success were statistically assessed, and opinions about directive implementation were documented. Results. Facilities experienced in manufacturing cell therapy transplant products are the most successful in developing ATMPs. New centres lacking this background struggle to enter the field, and there remains a shortage of facilities in academia participating in translational research. This is compounded by heterogeneous implementation of the regulations across MS. Conclusions. GMP facilities successfully developing ATMPs are present in all MS. However, the implementation of regulations is heterogeneous between MS, with substantial differences in the definition of ATMPs and in the approved manufacturing environment. The cost of GMP compliance is underestimated by research funding bodies. This is detrimental to development of new ATMPs and commercialization of any that are successful in early clinical trials. Academic GMP practitioners should strengthen their political visibility and contribute to the development of functional and effective European Union legislation in this field.},
  author       = {Pearce, Kim F. and Hildebrandt, Martin and Greinix, Hildegard and Scheding, Stefan and Koehl, Ulrike and Worel, Nina and Apperley, Jane and Edinger, Matthius and Hauser, Andrea and Mischak-Weissinger, Eva and Dickinson, Anne M. and Lowdell, Mark W.},
  issn         = {1477-2566},
  keyword      = {advanced therapy medicinal products,European Union,good manufacturing,practice,manufacturing,regulation},
  language     = {eng},
  number       = {3},
  pages        = {289--297},
  publisher    = {Taylor & Francis},
  series       = {Cytotherapy},
  title        = {Regulation of advanced therapy medicinal products in Europe and the role of academia},
  url          = {http://dx.doi.org/10.1016/j.jcyt.2013.08.003},
  volume       = {16},
  year         = {2014},
}