A literature review was conducted with the expression “Exempted Advanced Therapy Medicinal Products, in English and in French between the 8 October and 12 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
Abou-El-Enein, Mohamed, Ahmed Elsanhoury, and Petra Reinke. ‘Overcoming Challenges Facing Advanced Therapies in the EU Market’. Cell Stem Cell 19, no. 3 (1 September 2016): 293-297. https://doi.org/10.1016/j.stem.2016.08.012.
Aguilera-Cobos, Lorena, María Piedad Rosario-Lozano, Angela Ponce-Polo, Juan Antonio Blasco-Amaro, and David Epstein. ‘Barriers for the Evaluation of Advanced Therapy Medicines and Their Translation to Clinical Practice: Umbrella Review’. Health Policy 126, no. 12
(1 December 2022): 1248-55. https://doi.org/10.1016/j.healthpol.2022.10.007.
Ala-Mononen, Katri. ‘Biomaterials in the Manufacturing of Advanced Therapy Medicinal Products Used in Bone Replacement’. Tampere University, April 2022, 1-31.
Ancans, Jains. ‘Cell Therapy Medicinal Product Regulatory Framework in Europe and Its Application for MSC-Based Therapy Development’. Frontiers In Immunology 3 (2012): 253. https://doi.org/10.3389/fimmu.2012.00253.
Antonio Gomez-Outes a, Aranzazu Sancho-Lopez b., Antonio J. Carcas Sansuan c, and Cristina Avenda˜no-Sola b. ‘Clinical Research and Drug Regulation in the Challenging Times of Individualized Therapies: A Pivotal Role of Clinical Pharmacology’. Pharmacological Research 199 (1 January 2024): 107045. https://doi.org/10.1016/j.phrs.2023.107045.
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/jlb/lsac027
Buchholz, Christian J., Ralf Sanzenbacher, and Silke Schuele. ‘The European Hospital Exemption Clause-New Option for Gene Therapy?’ Human Gene Therapy 23, no. 1 (January 2012): 7-12. https://doi.org/10.1089/hum.2011.2529.
Ciccocioppo, Rachele, Patrizia Comoli, Giuseppe Astori, Francesca del Bufalo, Malvina Prapa, Massimo Dominici, and Franco Locatelli. ‘Developing Cell Therapies as Drug Products’. British Journal of Pharmacology 178, no. 2 (2021): 262-279. https://doi.org/10.1111/bph.15305.
Coppens, Delphi G. M., Helga Gardarsdottir, Marie L. De Bruin, Pauline Meij, Hubert G. M. Leufkens, and Jarno Hoekman. ‘Regulating Advanced Therapy Medicinal Products through the Hospital Exemption: An Analysis of Regulatory Approaches in Nine EU Countries’. Regenerative Medicine 15, no. 8 (August 2020): 2015-2028. https://doi.org/10.2217/rme-2020-0008.
Coppens, Delphi G. M., Jarno Hoekman, Marie L. De Bruin, Ineke C. M. Slaper-Cortenbach, Hubert G. M. Leufkens, Pauline Meij, and Helga Gardarsdottir. ‘Advanced Therapy Medicinal Product Manufacturing under the Hospital Exemption and Other Exemption Pathways in Seven European Union Countries’. Cytotherapy 22, no. 10 (1 October 2020): 592-600. https://doi.org/10.1016/j.jcyt.2020.04.092.
Corbett, Mark S., Andrew Webster, Robert Hawkins, and Nerys Woolacott. ‘Innovative Regenerative Medicines in the EU: A Better Future in Evidence?’ BMC Medicine 15, no. 1 (8 March 2017): 49. https://doi.org/10.1186/s12916-017-0818-4.
Cuende, Natividad, Christelle Boniface, Christopher Bravery, Miguel Forte, Rosaria Giordano, Martin Hildebrandt, Ander Izeta, and Massimo Dominici. ‘The Puzzling Situation of Hospital Exemption for Advanced Therapy Medicinal Products in Europe and Stakeholders’ Concerns’. Cytotherapy 16, no. 12 (1 December 2014): 1597-1600. https://doi.org/10.1016/j.jcyt.2014.08.007.
Cuende, Natividad, Rachele Ciccocioppo, Miguel Forte, Jacques Galipeau, Laertis Ikonomou, Bruce L. Levine, Alok Srivastava, and Patricia J. Zettler. ‘Patient Access to and Ethical Considerations of the Application of the European Union Hospital Exemption Rule for Advanced Therapy Medicinal Products’. Cytotherapy 24, no. 7 (1 July 2022): 686-690. https://doi.org/10.1016/j.jcyt.2022.03.007.
Cuende, Natividad, Concha Herrera, and Armand Keating. ‘When the Best Is the Enemy of the Good: The Case of Bone-Marrow Mononuclear Cells to Treat Ischemic Syndromes’. Haematologica 98, no. 3 (March 2013): 323–324. https://doi.org/10.3324/haematol.2013.086231.
Delgadillo, Joaquín, Erja Kerkelä, Allison Waters, Emile van den Akker, Chantal Lechanteur, Etienne Baudoux, Nicola Gardiner, John De Vos, and Joaquim Vives. ‘A Management Model in Blood, Tissue and Cell Establishments to Ensure Rapid and Sustainable Patient Access to Advanced Therapy Medicinal Products in Europe’. Cytotherapy 25, no. 12 (1 December 2023): 1259-1264. https://doi.org/10.1016/j.jcyt.2023.08.001.
Detela, Giulia, and Anthony Lodge. ‘EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation’. Molecular Therapy – Methods & Clinical Development 13 (14 June 2019): 205-232. https://doi.org/10.1016/j.omtm.2019.01.010.
Dulak, Józef, and Marlena Pecyna. ‘Unproven Cell Interventions in Poland and the Exploitation of European Union Law on Advanced Therapy Medicinal Products’. Stem Cell Reports 18, no. 8 (8 August 2023): 1610-1620. https://doi.org/10.1016/j.stemcr.2023.05.017.
Elsanhoury, Ahmed, Ralf Sanzenbacher, Petra Reinke, and Mohamed Abou-El-Enein. ‘Accelerating Patients’ Access to Advanced Therapies in the EU’. Molecular Therapy – Methods & Clinical Development 7 (15 December 2017): 15-19. https://doi.org/10.1016/j.omtm.2017.08.005.
Erben, Reinhold G., Beatriz Silva-Lima, Ilona Reischl, Gustav Steinhoff, Gudrun Tiedemann, Wilfried Dalemans, Alexander Vos, et al. ‘White Paper on How to Go Forward with Cell-Based Advanced Therapies in Europe’. Tissue Engineering. Part A 20, no. 19-20 (1 October 2014): 2549–2554. https://doi.org/10.1089/ten.tea.2013.0589.
Faulkner, Alex. ‘Commensuration and Proliferation: Similarity and Divergence in Law’s Shaping of Medical Technology’. Law, Innovation and Technology 4, no. 2 (2012): 165-184. https://doi.org/10.5235/LIT.4.2.165.
Faulkner, Alex. ‘Opening the Gateways to Market and Adoption of Regenerative Medicine? The UK Case in Context’. Regenerative Medicine 11, no. 3 (1 April 2016): 321-330. https://doi.org/10.2217/rme-2015-0046.
Fischer, Piret, Thomas Reiss, Jörg Mahlich, Erwan Gicquel, Heike Aichinger, Liliya Pullmann, and Tanja Bratan. ‘Unlocking the Value of Innovative Medicines: Insights from the Advanced Therapy Medicinal Products (ATMP) Innovation Systems in Germany and Sweden’. Health Policy and Technology 12, no. 2 (1 June 2023): 100744. https://doi.org/10.1016/j.hlpt.2023.100744.
Flory, Egbert, and Jens Reinhardt. ‘European Regulatory Tools for Advanced Therapy Medicinal Products’. Transfusion Medicine and Hemotherapy 40, no. 6 (29 October 2013): 409-412. https://doi.org/10.1159/000356364.
‘Focus Hospital Exemption on Developing Innovative and Safe Treatments for Patients’. Regenerative Medicine 8, no. 2 (1 March 2013): 121-123. https://doi.org/10.2217/rme.12.116.
Forgó, Nikolaus, and Martin Hildebrandt. ‘Joint Conference on the Impact of EU Legislation on Therapeutic Advance’. Cytotherapy 15, no. 12 (1 December 2013): 1444-1448. https://doi.org/10.1016/j.jcyt.2013.06.011.
Gerke, Sara, and Shaun D. Pattinson. ‘EU Marketing Authorisation of Orphan Medicinal Products and Its Impact on Related Research’. European Journal of Health Law 24, no. 5 (2017): 541-564. https://doi.org/DOI 10.1163/15718093-12341439.
Goula, Aspasia, Vasiliki Gkioka, Efstathios Michalopoulos, Michalis Katsimpoulas, Michel Noutsias, Eirini Faidra Sarri, Catherine Stavropoulos, and Alkiviadis Kostakis. ‘Advanced Therapy Medicinal Products Challenges and Perspectives in Regenerative Medicine’. Journal of Clinical Medicine Research 12, no. 12 (December 2020): 780-786. https://doi.org/10.14740/jocmr3964.
Hills, Allison, Jacquelyn Awigena-Cook, Ken Genenz, Mareike Ostertag, Simon Butler, Anne-Virginie Eggimann, and Annie Hubert. ‘An Assessment of the Hospital Exemption Landscape across European Member States: Regulatory Frameworks, Use and Impact’. Cytotherapy 22, no. 12 (1 December 2020): 772-779.e1. https://doi.org/10.1016/j.jcyt.2020.08.011.
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.
Hourd, Paul, Amit Chandra, Nick Medcalf, and David J. Williams. ‘Regulatory Challenges for the Manufacture and Scale-out of Autologous Cell Therapies’. In StemBook. Cambridge (MA): Harvard Stem Cell Institute, 2014. http://www.ncbi.nlm.nih.gov/books/NBK201975/.
Ivaskiene, Tatjana, Mykolas Mauricas, and Justinas Ivaska. ‘Hospital Exemption for Advanced Therapy Medicinal Products: Issue in Application in the European Union Member States’. Current Stem Cell Research & Therapy 12, no. 1 (2017): 45-51. https://doi.org/10.2174/1574888X11666160714114854.
Juan, Manel, Julio Delgado, Gonzalo Calvo, Esteve Trias, and Alvaro Urbano-Ispizua. ‘Is Hospital Exemption an Alternative or a Bridge to European Medicines Agency for Developing Academic Chimeric Antigen Receptor T-Cell in Europe? Our Experience with ARI-0001’. HUMAN GENE THERAPY 32, no. 19-20 (October 2021): 1004-1007. https://doi.org/10.1089/hum.2021.168.
Labedz-Maslowska, Anna, Agnieszka Szkaradek, Tomasz Mierzwinski, Zbigniew Madeja, and Ewa Zuba-Surma. ‘Processing and Ex Vivo Expansion of Adipose Tissue-Derived Mesenchymal Stem/Stromal Cells for the Development of an Advanced Therapy Medicinal Product for Use in Humans’. Cells 10, no. 8 (August 2021): 1908. https://doi.org/10.3390/cells10081908.
Lirsac, Pierre Noel, Olivier Blin, Jérémy Magalon, Pierre Angot, Estelle de Barbeyrac, Pascal Bilbault, Elisabeth Bourg, et al. ‘Creating Conditions for the Success of The French Industrial Advanced Therapy Sector’. Therapies, XXX Rencontres nationales de Pharmacologie et Recherche clinique, pour l’Innovation et l’Évaluation des Technologies de Santé Tables rondes Giens – 5 au 6 octobre 2014, 70, no. 1 (1 January 2015): 83-94. https://doi.org/10.2515/therapie/2015005.
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.
Mansnérus, Juli. ‘Encountering Challenges with the Eu Regulation on Advance Therapy Medical Products’. European Journal of Health Law 22, no. 5 (28 October 2015): 426-461. https://doi.org/10.1163/15718093-12341369.
Marton Varju and Judit Sandor. ‘Creating European Markets through Regulation: The Case of the Regulation on Advanced Therapy Medicinal Products | Westlaw UK’. European Law Review 41, no. 1 (2016): 25-43.
McMahon, Aisling. ‘Access to CAR-T Therapies in Ireland: Legal, Ethical & Broader Policy Considerations’. Maynooth University, 25 January 2024.
Megías-Vericat, Juan Eduardo, Ana Bonora-Centelles A, Tomás Palanques-Pastor, Cristóbal Eduardo Aguilar Gallardo, Manuel Guerreiro, Inés Gómez Seguí, Javier De La Rubia, and José Luis Poveda Andrés. ‘[Translated Article] New Challenges in Advanced Therapies’. Farmacia Hospitalaria, Nuevos roles y retos del farmacéutico de hospital, 48 (1 July 2024): TS21-27. https://doi.org/10.1016/j.farma.2024.05.001.
Nagpal, Anjali, Chris Juttner, Monica Anne Hamilton-Bruce, Paul Rolan, and Simon A. Koblar. ‘Stem Cell Therapy Clinical Research: A Regulatory Conundrum for Academia. Advanced Drug Delivery Reviews, Editors’, Collection 2017, 122 (1 December 2017): 105–114. https://doi.org/10.1016/j.addr.2016.10.001.
Olry de Labry-Lima, Antonio, Angela Ponce-Polo, Leticia García-Mochón, Marta Ortega-Ortega, Daniel Pérez-Troncoso, and David Epstein. ‘Challenges for Economic Evaluations of Advanced Therapy Medicinal Products: A Systematic Review’. Value in Health 26, no. 1 (1 January 2023): 138-150. https://doi.org/10.1016/j.jval.2022.07.004.
Pearce, Kim F., Martin Hildebrandt, Hildegard Greinix, Stefan Scheding, Ulrike Koehl, Nina Worel, Jane Apperley, et al. ‘Regulation of Advanced Therapy Medicinal Products in Europe and the Role of Academia’. Cytotherapy 16, no. 3 (1 March 2014): 289-297. https://doi.org/10.1016/j.jcyt.2013.08.003.
Pirnay, Jean-Paul, Alain Vanderkelen, Daniel De Vos, Jean-Pierre Draye, Thomas Rose, Carl Ceulemans, Nadine Ectors, Isabelle Huys, Serge Jennes, and Gilbert Verbeken. ‘Business Oriented EU Human Cell and Tissue Product Legislation Will Adversely Impact Member States’ Health Care Systems’. Cell and Tissue Banking 14, no. 4 (1 December 2013): 525-560. https://doi.org/10.1007/s10561-013-9397-6.
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.
Raposo, Vera Lúcia. ‘A Room with a View (and with a Gene Therapy Drug): Gene Therapy Medicinal Products and Genetic Tourism in Europe’. European Journal of Health Law 29, no. 3–5 (8 April 2022): 504-520. https://doi.org/10.1163/15718093-bja10083.
Rigter, Tessel, David Klein, Stephanie S. Weinreich, and Martina C. Cornel. ‘Moving Somatic Gene Editing to the Clinic: Routes to Market Access and Reimbursement in Europe’. European Journal of Human Genetics 29, no. 10 (October 2021): 1477-1484. https://doi.org/10.1038/s41431-021-00877-y.
Salvatore, Vincenzo. ‘Exploring Solutions to Foster ATMP Development and Access to Patients in Europe Special Issue: Innovative Medicine and Research: Ethical, Legal and Regulatory Issues’. European Journal of Health Law 27, no. 3 (2020): 259-273. https://doi.org/DOI:10.1163/15718093-BJA10016.
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.
Taboulet, Florence. ‘Les médicaments de thérapie innovante : quelles spécificités en droit pharmaceutique ?’ Quaderni. Communication, technologies, pouvoir, no. 81 (5 June 2013): 15-27. https://doi.org/10.4000/quaderni.702.
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 (1 February 2022): 156-159. https://doi.org/10.1038/s41409-021-01463-y.
Van Wilder, Philippe. ‘Advanced Therapy Medicinal Products and Exemptions to the Regulation 1394/2007: How Confident Can We Be? An Exploratory Analysis’. Frontiers in Pharmacology 3 (14 February 2012). https://doi.org/10.3389/fphar.2012.00012.
Verbeken, Gilbert, Jean-Paul Pirnay, Rob Lavigne, Serge Jennes, Daniel De Vos, Minne Casteels, and Isabelle Huys. ‘Call for a Dedicated European Legal Framework for Bacteriophage Therapy’. Archivum Immunologiae et Therapiae Experimentalis 62, no. 2 (1 April 2014): 117-129. https://doi.org/10.1007/s00005-014-0269-y.
Vives, Joaquim, Irene Oliver-Vila, and Arnau Pla. ‘Quality Compliance in the Shift from Cell Transplantation to Cell Therapy in Non-Pharma Environments’. Cytotherapy 17, no. 8 (1 August 2015): 1009-1014. https://doi.org/10.1016/j.jcyt.2015.02.002.
Wilde, Sofieke de, Louise Veltrop-Duits, Merel Hoozemans-Strik, Thirza Ras, Janine Blom-Veenman, Henk-Jan Guchelaar, Maarten Zandvliet, and Pauline Meij. ‘Hurdles in Clinical Implementation of Academic Advanced Therapy Medicinal Products: A National Evaluation’. Cytotherapy 18, no. 6 (1 June 2016): 797-805. https://doi.org/10.1016/j.jcyt.2016.02.010.
