Clinical Development and Commercialization of Advanced Therapy Medicinal Products in the European Union: How Are the Product Pipeline and Regulatory Framework Evolving?

Human Gene Therapy. Clinical Development
Tomáš BoráňPaula Salmikangas

Abstract

The research and development of advanced therapy medicinal products (ATMPs) has been active in Europe and worldwide during recent years. Yet, the number of licensed products remains low. The main expected legal change in the near future in the European Union (EU) concerns the regulation on clinical trials (536/2014), which will come into force in 2018. With this new framework, a more harmonized and swift process for approval of clinical trials is anticipated, which is expected to support the entry of new innovations into the EU market. A survey on ATMPs in clinical trials during 2010-2015 in the EU was conducted in order to study the trends of ATMP development since the earlier survey published in 2012. According to the results, the number of clinical trials using ATMPs is slowly increasing in the EU. Yet, the focus is still in early development, and the projects are mainly carried out by small and medium-sized enterprises, academia, and hospitals. Oncology is the main area of clinical development. Yet, the balance between cell-based products and gene therapy medicinal products in this area may be changing in the future due to the new T-cell technologies. Many limitations and challenges are identified for ATMP development, requ...Continue Reading

References

Apr 7, 2011·Nature Reviews. Genetics·Mark A Kay
Mar 2, 2012·Molecular Therapy : the Journal of the American Society of Gene Therapy·Romaldas MaciulaitisChristian K Schneider
Jan 29, 2013·The Journal of Gene Medicine·Samantha L GinnJo Wixon
Apr 4, 2015·Regenerative Medicine·Mahendra RaoSusan Solomon
Oct 2, 2015·Cell Transplantation·Tiziana SquillaroUmberto Galderisi
Dec 25, 2015·Proceedings. Biological Sciences·Mary Collins, Adrian Thrasher
Jan 23, 2016·Human Gene Therapy·Marina CavazzanaSalima Hacein-Bey-Abina
Feb 9, 2017·Stem Cells Translational Medicine·Arnold I CaplanBrock Reeve

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Citations

May 23, 2019·Tissue Engineering. Part a·Alicia J El Haj
Feb 28, 2020·Regenerative Medicine·Delphi Gm CoppensJarno Hoekman
Jul 14, 2018·BioMed Research International·Roxana-Maria AmărandiJorge S Burns
Feb 7, 2020·Bone Marrow Transplantation·Joaquim VivesNúria Nogués
Jun 19, 2018·Frontiers in Medicine·Cécile F RousseauGopalan Narayanan
May 28, 2019·Frontiers in Bioengineering and Biotechnology·Benjamen T O'DonnellBruce A Bunnell
Nov 10, 2018·Stem Cell Research & Therapy·Erika KleidermanBartha Maria Knoppers
Dec 12, 2020·Journal of Pharmaceutical Sciences·Dhananjay JereHanns-Christian Mahler
Dec 18, 2020·Frontiers in Surgery·Thomas H JovicIain S Whitaker
Jan 6, 2021·Cell and Tissue Banking·Francisco J Verdugo-AvelloSoraya Gutierrez
Feb 23, 2021·Frontiers in Cardiovascular Medicine·Paloma GastelurrutiaCarolina Gálvez-Montón
Aug 31, 2021·Transplant International : Official Journal of the European Society for Organ Transplantation·Emanuele CozziUNKNOWN for ESOT Workstream 1 of the TLJ (Transplantation Learning Journey) Project

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Software Mentioned

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