A literature review was conducted with the expressions “CAR-T cells” and “Regulation” and “European Union”, in English and in French between the 18 and 23 November 2024, in the following databases: Dalloz, HeinOnline, Lexbase, Lexis Nexis, Lextenso, Navis, Doctrinal Plus, Westlaw UK, Brill and Cairn, Bibliothèque numérique en droit de la santé, Wiley Online Library, Lamyline Reflex, Cambridge University Press Revues – Archives ISTEX, De Gruyter revues – Archives ISTEX, Encyclopédie Max Planck de droit international public, JSTOR, Law Review Commons, OpenEdition Journals, ScienceDirect, Springer Nature, Web of Science, Google Scholar and PubMed.
Please find below the most relevant results of this review
Abinader, Luis Gil, and Jorge L. Contreras. ‘The Patentability of Genetic Therapies: CAR-T and Medical Treatment Exclusions around the World Articles from the Fifth Global Congress on Intellectual Property and the Public Interest’. American University International Law Review 34, no. 4 (2019 2018): 705-762.
Arnaudo, Luca. ‘On CAR-Ts, Decentralized in-House Models, and the Hospital Exception. Routes for Sustainable Access to Innovative Therapies’. Journal of Law and the Biosciences 9, no. 2 (2022): 1-19. https://doi.org/10.1093/lb/sacO27.
Bellino, Stefania, Anna La Salvia, Maria Francesca Cometa, and Rosanna Botta. ‘Cell-Based Medicinal Products Approved in the European Union: Current Evidence and Perspectives’. Frontiers in Pharmacology 14 (31 July 2023). https://doi.org/10.3389/fphar.2023.1200808.
Berquier, Maxime, and Nicolas Boissel. ‘La coordination du parcours patient lors d’un traitement par CAR-T cells’. Bulletin Infirmier du Cancer 19, no. 4 (2019): 152-160. https://doi.org/10.1684/bic.2019.87.
Brigand, Théau, Olivier Maguet, Juliana Veras, and Patrick Durisch. ‘Plaidoyer pour l’accès équitable aux nouvelles technologies de santé : le cas du Kymriah®’. Sante Publique Vol. 32, no. 5 (2020): 525-529.
Buechner, Jochen, Marie José Kersten, HOVON/LLPC (Lunenburg Lymphoma Phase I/II Consortium) working Group, Miriam Fuchs, Florence Salmon, and Ulrich Jäger. ‘Chimeric Antigen Receptor-T Cell Therapy: Practical Considerations for Implementation in Europe’. HemaSphere 2, no. 1 (2018): e18. https://doi.org/10.1097/HS9.0000000000000018.
Cauchon, Nina S., Shirley Oghamian, Soraya Hassanpour, and Michael Abernathy. ‘Innovation in Chemistry, Manufacturing, and Controls—A Regulatory Perspective From Industry’. Journal of Pharmaceutical Sciences 108, no. 7 (1 July 2019): 207-237. –h2ttps://doi.org/10.1016/j.xphs.2019.02.007.
Celis, Patrick, Giada Farinelli, Ana Hidalgo-Simon, Pauline Meij, Mara Tihaya, Martina Schüssler-Lenz, and Marcos Timón. ‘EMA Commentary on the Guideline on Quality, Nonclinical and Clinical Aspects of Medicinal Products Containing Genetically Modified Cells’. British Journal of Clinical Pharmacology 90, no. 5 (2024): 1203-1212. https://doi.org/10.1111/bcp.16047.
Chabannon, C., O. Caunday-Rigot, C. Faucher, I. Slaper-Cortenbach, B. Calmels, C. Lemarie, A. Mahalatchimy, E. McGrath, and E. Rial-Sebbag. ‘Accreditation and Regulations in Cell Therapy’. ISBT Science Series 11, no. S1 (2016): 271-276. https://doi.org/10.1111/voxs.12205.
Chabannon, Christian. ‘Thérapies cellulaires et géniques : la guérison à l’horizon ?’ Cancer(s) et psy(s) 5, no. 1 (2020): 122-128. https://doi.org/10.3917/crpsy.005.0122.
Chabannon, Christian, and Jérôme Larghero. ‘Prise en charge des cellules CAR-T au sein des établis- sements de santé français : fabrication, distribution, et aspects réglementaires’. Bulletin de l’Académie Nationale de Médecine 202, no. 7 (1 September 2018): 1431–1440. https://doi.org/10.1016/S0001-4079(19)30209-2.
Chabannon, Christian, and Jérôme Larghero. ‘Réglementations applicables aux CAR-T cells : comment les établissements de santé français peuvent-ils s’organiser pour participer à la production et permettre la délivrance de ces immunothérapies innovantes ?’ Bulletin du Cancer, Les cellules CAR-T : une révolution thérapeutique ?, 105 (1 December 2018): S198-204. https://doi.org/10.1016/S0007-4551(19)30050-5.
Das, Amritava, Krishanu Saha, and Pilar N. Ossorio. ‘Fairness in Manufacturing Cellular Therapies Open Peer Commentaries’. American Journal of Bioethics 18, no. 4 (2018): 68-70. https://doi.org/10.1080/15265161.2018.1445792.
De Santis, Gil Cunha. ‘Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on Genetically Modified Cells. Special Article: Compassionate Use and Clinical Trial on CAR-T Cells’. Hematology, Transfusion and Cell Therapy 43 (1 November 2021): S64-567. https://doi.org/10.1016/j.htct.2021.09.009.
Elsallab, Magdi, and Marcela V. Maus. ‘Expanding Access to CAR T Cell Therapies through Local Manufacturing’. Nature Biotechnology 41, no. 12 (1 December 2023): 1698-1708. -1 https://doi.org/10.1038/s41587-023-01981-8.
Fritsche, Enrico, Magdi Elsallab, Michaela Schaden, Spencer Hey, and Mohamed Abou-el-Enein. ‘Post-Marketing Safety and Efficacy Surveillance of Cell and Gene Therapies in the EU: A Critical Review’. Cell and Gene Therapy Insights 5 (15 November 2019). https://doi.org/10.18609/cgti.2019.156.
Gee, Adrian P. ‘GMP CAR-T Cell Production’. Best Practice & Research Clinical Haematology, CAR T Cells, Immunologic and Cellular Therapies in Hematologic Malignancies, 31, no. 2 (1 June 2018): 126-134. https://doi.org/10.1016/j.beha.2018.01.002.
Giorgioni, Lorenzo, Alessandra Ambrosone, Maria Francesca Cometa, Anna Laura Salvati, and Armando Magrelli. ‘CAR-T State of the Art and Future Challenges, A Regulatory Perspective’. International Journal of Molecular Sciences 24, no. 14 (January 2023): 11803. https://doi.org/10.3390/ijms241411803.
Gou, Lijuan, Jianchao Gao, Huan Yang, and Chenyan Gao. ‘The Landscape of CAR T-Cell Therapy in the United States and China: A Comparative Analysis’. International Journal of Cancer 144, no. 8 (2019): 2043-2050. https://doi.org/10.1002/ijc.31924.
Hartmann, Jessica, Martina Schüßler‐Lenz, Attilio Bondanza, and Christian J Buchholz. ‘Clinical Development of CAR T Cells—Challenges and Opportunities in Translating Innovative Treatment Concepts’. EMBO Molecular Medicine 9, no. 9 (September 2017): 1183–1197. https://doi.org/10.15252/emmm.201607485.
Hirai, Takamasa, Satoshi Yasuda, Akihiro Umezawa, and Yoji Sato. ‘Country-Specific Regulation and International Standardization of Cell-Based Therapeutic Products Derived from Pluripotent Stem Cells’. Stem Cell Reports 18, no. 8 (8 August 2023): 1573-1591. https://doi.org/10.1016/j.stemcr.2023.05.003.
Holzinger, Astrid, and Hinrich Abken. ‘Treatment with Living Drugs: Pharmaceutical Aspects of CAR T Cells’. Pharmacology 107, no. 9–10 (13 June 2022): 446-463. https://doi.org/10.1159/000525052.
Hort, Simon, Carmen Sanges, John J. L. Jacobs, Michael Hudecek, and Robert H. Schmitt. ‘Digital Transformation of CAR-T Cell Therapy – Challenges and Potential for Industry 4.0’. Procedia CIRP, 56th CIRP International Conference on Manufacturing Systems 2023, 120 (1 January 2023): 1034-1040. https://doi.org/10.1016/j.procir.2023.09.121.
Joy, R., K. Phair, R. O’Hara, and D. Brady. ‘Recent Advances and Current Challenges in CAR-T Cell Therapy’. Biotechnology Letters 46, no. 1 (1 February 2024): 115-126. https://doi.org/10.1007/s10529-023-03461-0.
Kalland, Maria Elisabeth, Tomas Pose-Boirazian, Gloria Maria Palomo, Frauke Naumann-Winter, Enrico Costa, Darius Matusevicius, Dinah M. Duarte, et al. ‘Advancing Rare Disease Treatment: EMA’s Decade-Long Insights into Engineered Adoptive Cell Therapy for Rare Cancers and Orphan Designation’. Gene Therapy 31, no. 7 (1 July 2024): 366–377. https://doi.org/10.1038/s41434-024-00446-0.
Kim, Jiyeon. ‘Patent Infringement in Personalized Medicine: Limitations of the Existing Exemption Mechanisms Notes’. Washington University Law Review 96, no. 3 (2019 2018): 623-648.
Komanduri, Shraddha. ‘Regulation of CAR-T Cell Therapies – The Past, Present, and Future’. FDLI Update 2020, no. 4 (2020): 52-61.
Krackhardt, Angela M., Brigitte Anliker, Martin Hildebrandt, Michael Bachmann, Stefan B. Eichmüller, Dirk M. Nettelbeck, Matthias Renner, et al. ‘Clinical Translation and Regulatory Aspects of CAR/TCR-Based Adoptive Cell Therapies—the German Cancer Consortium Approach’. Cancer Immunology, Immunotherapy : CII 67, no. 4 (29 January 2018): 513. https://doi.org/10.1007/s00262-018-2119-y.
Laramy, Christine R. O’Brien. ‘The CAR-T Cell Therapy Innovation Drivers: A Yescarta Case Study Life Science Innovation Case Study Project’. Berkeley Technology Law Journal 39, no. Special Issue (2024): 553-612. https://doi.org/10.15779/Z38CZ32610.
Levine, Bruce L., James Miskin, Keith Wonnacott, and Christopher Keir. ‘Global Manufacturing of CAR T Cell Therapy’. Molecular Therapy – Methods & Clinical Development 4 (17 March 2017): 92-101. https://doi.org/10.1016/j.omtm.2016.12.006.
Litvinova, Yulia, Sherry Merkur, Sara Allin, Ester Angulo-Pueyo, Daiga Behmane, Enrique Bernal-Delgado, Miriam Dalmas, et al. ‘Availability and Financing of CAR-T Cell Therapies: A Cross-Country Comparative Analysis’. Health Policy 149 (1 November 2024): 105153. https://doi.org/10.1016/j.healthpol.2024.105153.
McGrath, Eoin, and Petr Machalik. ‘The Regulatory Framework for CAR-T Cells in Europe: Current Status and Foreseeable Changes AND Centre Qualification by Competent Authorities and Manufacturers’. In The EBMT/EHA CAR-T Cell Handbook, edited by Nicolaus Kröger, John Gribben, Christian Chabannon, Ibrahim Yakoub-Agha, and Hermann Einsele. Cham (CH): Springer, 2022. http://www.ncbi.nlm.nih.gov/books/NBK584158/.
Mcmahon, Aisling. ‘Access to CAR-T Therapies in Ireland: Legal, Ethical & Broader Policy Considerations’. Maynooth University, 18 October 2023, 1-96.
Mebarki, Miryam, Isabelle Madelaine, Jérôme Larghero, and Romain de Jorna. ‘Advanced Therapy Medicinal Products: Regulatory Framework, Hospital and Pharmaceutical Circuits in Europe and France’. Therapies, Hemato-oncopharmacology, 77, no. 2 (1 March 2022): 185-190. https://doi.org/10.1016/j.therap.2021.11.005.
Orentas, Rimas J., Boro Dropulić, and Marcos de Lima. ‘Place of Care Manufacturing of Chimeric Antigen Receptor Cells: Opportunities and Challenges’. Seminars in Hematology, Cell Therapy, 60, no. 1 (1 January 2023): 20-24. https://doi.org/10.1053/j.seminhematol.2023.01.001.
Overbeeke, Eline van, Sissel Michelsen, Mondher Toumi, Hilde Stevens, Mark Trusheim, Isabelle Huys, and Steven Simoens. ‘Market Access of Gene Therapies across Europe, USA, and Canada: Challenges, Trends, and Solutions’. Drug Discovery Today 26, no. 2 (1 February 2021): 399-415. https://doi.org/10.1016/j.drudis.2020.11.024.
Paubel, Pascal. ‘Médicaments à l’hôpital : une régulation complexe pour une dépense croissante’, 2020. https://www.dalloz.fr/documentation/Document?id=RDSS/DOSS/2020/0434.
Poveda-Andrés, José Luis, María José Ruiz-Caldes, María José Carreras-Soler, Ana Clopés-Estela, Sandra Flores-Moreno, Javier García-Pellicer, Míriam Giráldez-Quiroga, et al. ‘Pharmacotherapeutic management of advanced therapy medicinal products’. Farmacia Hospitalaria 46, no. 2 (1 March 2022): 88-95. https://doi.org/10.7399/fh.13036.
Priesner, Christoph, and Martin Hildebrandt. ‘Advanced Therapy Medicinal Products and the Changing Role of Academia’. Transfusion Medicine and Hemotherapy 49, no. 3 (16 May 2022): 158-162. https://doi.org/10.1159/000524392.
Prod’homme, Chloé. ‘Justice distributive et accès à l’innovation : questionnement éthique autour des cellules T à récepteur antigénique chimérique’. Hématologie 25, no. 2 (2019): 100–104. https://doi.org/10.1684/hma.2019.1422.
Rial-Sebbag, Emmanuelle, and Christian Chabannon. ‘Enjeux juridiques et pour le système de santé du développement d’une nouvelle classe de médicaments de thérapie innovante en onco-immunologie : les “Car-T Cells”’. Journal international de bioethique et d’ethique des sciences 29, no. 2 (27 July 2018): 113-128².
Roddie, Claire, Maeve O’Reilly, Juliana Dias Alves Pinto, Ketki Vispute, and Mark Lowdell. ‘Manufacturing Chimeric Antigen Receptor T Cells: Issues and Challenges’. Cytotherapy 21, no. 3 (1 March 2019): 327-340. https://doi.org/10.1016/j.jcyt.2018.11.009.
S s, Kirthiga Devi, Ramesh Joga, Saurabh Srivastava, Kalpana Nagpal, Isha Dhamija, Parul Grover, and Sandeep Kumar. ‘Regulatory Landscape and Challenges in CAR-T Cell Therapy Development in the US, EU, Japan, and India’. European Journal of Pharmaceutics and Biopharmaceutics 201 (1 August 2024): 114361. https://doi.org/10.1016/j.ejpb.2024.114361.
Sanchez-Guijo, Fermin, Joaquim Vives, Annalisa Ruggeri, Christian Chabannon, Selim Corbacioglu, Harry Dolstra, Dominique Farge, et al. ‘Current Challenges in Cell and Gene Therapy: A Joint View from the European Committee of the International Society for Cell & Gene Therapy (ISCT) and the European Society for Blood and Marrow Transplantation (EBMT)’. Cytotherapy 26, no. 7 (1 July 2024): 681-685. https://doi.org/10.1016/j.jcyt.2024.02.007.
Seimetz, Diane, Karl Heller, and Jan Richter. ‘Approval of First CAR-Ts: Have We Solved All Hurdles for ATMPs?’ Cell Medicine 11 (1 January 2019): 2155179018822781. https://doi.org/10.1177/2155179018822781.
Sharma, Pinky, Vikas Jhawat, Pooja Mathur, and Rohit Dutt. ‘Innovation in Cancer Therapeutics and Regulatory Perspectives’. Medical Oncology 39, no. 5 (23 February 2022): 76. https://doi.org/10.1007/s12032-022-01677-0.
Trias, Esteve, Manel Juan, Alvaro Urbano-Ispizua, and Gonzalo Calvo. ‘The Hospital Exemption Pathway for the Approval of Advanced Therapy Medicinal Products: An Underused Opportunity? The Case of the CAR-T ARI-0001’. Bone Marrow Transplantation 57, no. 2 (February 2022): 156-159. https://doi.org/10.1038/s41409-021-01463-y.
Vucinic, Vladan, Andrea Quaiser, Philipp Lückemeier, Stephan Fricke, Uwe Platzbecker, and Ulrike Koehl. ‘Production and Application of CAR T Cells: Current and Future Role of Europe’. Frontiers in Medicine 8 (16 August 2021). https://doi.org/10.3389/fmed.2021.713401.
Wang, Valentine, Mélanie Gauthier, Véronique Decot, Loïc Reppel, and Danièle Bensoussan. ‘Systematic Review on CAR-T Cell Clinical Trials Up to 2022: Academic Center Input’. Cancers 15, no. 4 (January 2023): 1003. https://doi.org/10.3390/cancers15041003.
Wilkerson, John. ‘Lawmakers Parrot Drug Makers’ Arguments For Wider CAR-T Coverage’. Inside CMS 22, no. 20 (16 May 2019): 25-25.
