Accelerating Patients' Access to Advanced Therapies in the EU

Overview

Journal Mol Ther Methods Clin Dev

Publisher Cell Press

Date 2017 Oct 4

PMID 28971109

Citations 9

Authors

Ahmed Elsanhoury

Ralf Sanzenbacher

Petra Reinke

Mohamed Abou-El-Enein

Affiliations

Soon will be listed here.

Citing Articles

The Potential of Mesenchymal Stem/Stromal Cells in Diabetic Wounds and Future Directions for Research and Therapy-Is It Time for Use in Everyday Practice?.

Sienko D, Szablowska-Gadomska I, Nowak-Szwed A, Rudzinski S, Gofron M, Zygmunciak P Int J Mol Sci. 2024; 25(22).

PMID: 39596237 PMC: 11594847. DOI: 10.3390/ijms252212171.

Expert Consideration on Regulatory Aspects for Perinatal Derivatives in Clinical Settings.

Hofmann N, Lafarge X, Antica M, Ferry N, Girandon L, Gramignoli R Stem Cells Transl Med. 2023; 12(5):258-265.

PMID: 37027834 PMC: 10184691. DOI: 10.1093/stcltm/szad017.

RESTORE Survey on the Public Perception of Advanced Therapies and ATMPs in Europe-Why the European Union Should Invest More!.

Goldsobel G, von Herrath C, Schlickeiser S, Brindle N, Stahler F, Reinke P Front Med (Lausanne). 2021; 8:739987.

PMID: 34765617 PMC: 8576137. DOI: 10.3389/fmed.2021.739987.

Advanced Therapy Medicinal Products Challenges and Perspectives in Regenerative Medicine.

Goula A, Gkioka V, Michalopoulos E, Katsimpoulas M, Noutsias M, Sarri E J Clin Med Res. 2021; 12(12):780-786.

PMID: 33447311 PMC: 7781285. DOI: 10.14740/jocmr3964.

Two Decades of Global Progress in Authorized Advanced Therapy Medicinal Products: An Emerging Revolution in Therapeutic Strategies.

Ramezankhani R, Torabi S, Minaei N, Madani H, Rezaeiani S, Hassani S Front Cell Dev Biol. 2021; 8:547653.

PMID: 33392179 PMC: 7773756. DOI: 10.3389/fcell.2020.547653.

References

Sergeeva A, Kolonin M, Molldrem J, Pasqualini R, Arap W . Display technologies: application for the discovery of drug and gene delivery agents. Adv Drug Deliv Rev. 2006; 58(15):1622-54. PMC: 1847402. DOI: 10.1016/j.addr.2006.09.018. View

Cuende N, Boniface C, Bravery C, Forte M, Giordano R, Hildebrandt M . The puzzling situation of hospital exemption for advanced therapy medicinal products in Europe and stakeholders' concerns. Cytotherapy. 2014; 16(12):1597-600. DOI: 10.1016/j.jcyt.2014.08.007. View

Renner M, Anliker B, Sanzenbacher R, Schuele S . Regulation of Clinical Trials with Advanced Therapy Medicinal Products in Germany. Adv Exp Med Biol. 2015; 871:87-101. DOI: 10.1007/978-3-319-18618-4_5. View

Santos R, Ursu O, Gaulton A, Bento A, Donadi R, Bologa C . A comprehensive map of molecular drug targets. Nat Rev Drug Discov. 2016; 16(1):19-34. PMC: 6314433. DOI: 10.1038/nrd.2016.230. View

Buchholz C, Sanzenbacher R, Schule S . The European hospital exemption clause-new option for gene therapy?. Hum Gene Ther. 2012; 23(1):7-12. DOI: 10.1089/hum.2011.2529. View

Feigal E, Tsokas K, Viswanathan S, Zhang J, Priest C, Pearce J . Proceedings: international regulatory considerations on development pathways for cell therapies. Stem Cells Transl Med. 2014; 3(8):879-87. PMC: 4116253. DOI: 10.5966/sctm.2014-0122. View

Schneider C, Salmikangas P, Jilma B, Flamion B, Todorova L, Paphitou A . Challenges with advanced therapy medicinal products and how to meet them. Nat Rev Drug Discov. 2010; 9(3):195-201. DOI: 10.1038/nrd3052. View

Gammie T, Lu C, Babar Z . Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries. PLoS One. 2015; 10(10):e0140002. PMC: 4599885. DOI: 10.1371/journal.pone.0140002. View

Yla-Herttuala S . The need for increased clarity and transparency in the regulatory pathway for gene medicines in the European Union. Mol Ther. 2012; 20(3):471-2. PMC: 3293610. DOI: 10.1038/mt.2012.14. View

10.

Abou-El-Enein M, Bauer G, Medcalf N, Volk H, Reinke P . Putting a price tag on novel autologous cellular therapies. Cytotherapy. 2016; 18(8):1056-1061. DOI: 10.1016/j.jcyt.2016.05.005. View

11.

Abou-El-Enein M, Bauer G, Reinke P, Renner M, Schneider C . A roadmap toward clinical translation of genetically-modified stem cells for treatment of HIV. Trends Mol Med. 2014; 20(11):632-42. DOI: 10.1016/j.molmed.2014.08.004. View

12.

Fesnak A, June C, Levine B . Engineered T cells: the promise and challenges of cancer immunotherapy. Nat Rev Cancer. 2016; 16(9):566-81. PMC: 5543811. DOI: 10.1038/nrc.2016.97. View

13.

Dominici M, Nichols K, Srivastava A, Weiss D, Eldridge P, Cuende N . Positioning a Scientific Community on Unproven Cellular Therapies: The 2015 International Society for Cellular Therapy Perspective. Cytotherapy. 2015; 17(12):1663-6. DOI: 10.1016/j.jcyt.2015.10.007. View

14.

Abou-El-Enein M, Elsanhoury A, Reinke P . Overcoming Challenges Facing Advanced Therapies in the EU Market. Cell Stem Cell. 2016; 19(3):293-7. DOI: 10.1016/j.stem.2016.08.012. View

15.

Loda M . Challenging Roadblocks to Cancer Cure. Cancer Res. 2016; 76(17):4924-30. DOI: 10.1158/0008-5472.CAN-16-1443. View

16.

Sanzenbacher R, Dwenger A, Schuessler-Lenz M, Cichutek K, Flory E . European regulation tackles tissue engineering. Nat Biotechnol. 2007; 25(10):1089-91. DOI: 10.1038/nbt1007-1089. View