A bibliographic research was conducted with the expressions “SME Office” and “SME Status”, in English and in French from 4 to 11 March 2025, in the following databases: Dalloz, HeinOnline, Lexbase, Lexis 360, Lextenso, Navis, Le Doctrinal, Brill revues – Archives ISTEX, Cairn revues, 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:
Amaouche, Nadia, Hélène Casaert Salomé, Olivier Collignon, Mariana Roldao Santos, and Constantinos Ziogas. ‘Marketing Authorisation Applications Submitted to the European Medicines Agency by Small and Medium-Sized Enterprises: An Analysis of Major Objections and Their Impact on Outcomes’. Drug Discovery Today 23, no. 10 (1 October 2018): 1801–1805.
https://doi.org/10.1016/j.drudis.2018.06.018
Bayon, Y., M. Bohner, D. Eglin, P. Procter, R. G. Richards, J. Weber, and D. I. Zeugolis. ‘Innovating in the Medical Device Industry – Challenges & Opportunities ESB 2015 Translational Research Symposium’. Journal of Materials Science: Materials in Medicine 27, no. 9 (1 September 2016): 1–7.
https://doi.org/10.1007/s10856-016-5759-5
Bird & Bird. ‘Legal And Regulatory Update’. Journal of Commercial Biotechnology 16, no. 2 (1 April 2010): 174–182.
https://doi.org/10.1057/jcb.2009.39
Bouwman, Luísa, Bruno Sepodes, Hubert Leufkens, and Carla Torre. ‘Trends in Orphan Medicinal Products Approvals in the European Union between 2010–2022’. Orphanet Journal of Rare Diseases 19, no. 1 (December 2024): 1–16.
https://doi.org/10.1186/s13023-024-03095-z
Brévignon-Dodin, Laure, and Pawanbir Singh. ‘ATMP in Practice: Towards a New Industry Landscape in Tissue Engineering’. Journal of Commercial Biotechnology 15, no. 1 (1 January 2009): 59–65.
https://doi.org/10.1057/jcb.2008.32
Bruttomesso, G., and N. Miglino. ‘Talking About…’. Journal of Thrombosis and Haemostasis 4, no. 3 (1 March 2006): 712–714.
https://doi.org/10.1111/j.1538-7836.2006.01863.x
Carr, M. ‘The Small- and Medium-Sized Enterprises Office (SME Office) at the European Medicines Agency’. Bundesgesundheitsblatt – Gesundheitsforschung – Gesundheitsschutz 53, no. 1 (1 January 2010): 20–23.
https://doi.org/10.1007/s00103-009-0989-z
Carvalho, Marta, Ana Paula Martins, and Bruno Sepodes. ‘Hurdles in Gene Therapy Regulatory Approval: A Retrospective Analysis of European Marketing Authorization Applications’. Drug Discovery Today 24, no. 3 (1 March 2019): 823–828.
https://doi.org/10.1016/j.drudis.2018.12.007
Cremer, Lara. ‘La recherche à l’école doctorale – Changement de paradigme dans l’innovation pharmaceutique et la distribution officinale. Analyse comparative des droits français, allemand et suisse⁎ – Commentaire par Mme Lara Cramer – Lexis 360 Intelligence’. RDJA, no. n° 4 (1 December 2021): 119–138.
Davydiuk, O. M., G. M. Shovkoplias, V. O. Usatyi, H. S. Ivanova, O. Yu Bytiak, and T. Duiunova. ‘Innovation in the Legal Regulation of Small (Medium) Enterprises in the Eu Law’. Science and Innovation 21, no. 1 (2025): 35–49.
https://doi.org/10.15407/scine21.01.035
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
Fürst-Ladani, Shayesteh, Anja Bührer, Walter Fürst, and Nathalie Schober-Ladani. ‘Regulatory Aspects for Approval of Advanced Therapy Medicinal Products in the EU’. In Drug Delivery and Targeting, 367–387. Springer, Cham, 2023.
https://doi.org/10.1007/164_2023_648
Garsen, Marjolein, Maaike Steenhof, and Alex Zwiers. ‘A Decade of Marketing Authorization Applications of Anticancer Drugs in the European Union: An Analysis of Procedural Timelines’. Therapeutic Innovation & Regulatory Science 55, no. 4 (1 July 2021): 633–642.
https://doi.org/10.1007/s43441-021-00260-5
Horst, Judith P. ter, Sada L. Turimella, Frans Metsers, and Alex Zwiers. ‘Implementation of Quality by Design (QbD) Principles in Regulatory Dossiers of Medicinal Products in the European Union (EU) Between 2014 and 2019’. Therapeutic Innovation & Regulatory Science 55, no. 3 (1 May 2021): 583–590.
https://doi.org/10.1007/s43441-020-00254-9
Jilma, Bernd. ‘Development of Advanced Therapy Medicinal Products — a Case for Early Scientific Advice’. In Clinical Pharmacology: Current Topics and Case Studies, 323–334. Springer, Vienna, 2010.
https://doi.org/10.1007/978-3-7091-0144-5_21
Klug, Bettina, Patrick Celis, Melanie Carr, and Jens Reinhardt. ‘Chapter Seventeen – Regulatory Structures for Gene Therapy Medicinal Products in the European Union’. In Methods in Enzymology, edited by Theodore Friedmann, 507:337–354. Gene Transfer Vectors for Clinical Application. Academic Press, 2012.
https://doi.org/10.1016/B978-0-12-386509-0.00017-X
Kondo, Hideyuki, Masayoshi Shibatsuji, and Naoyuki Yasuda. ‘Regulatory/Scientific Supports for Micro-, Small-, and Medium-Sized Enterprises (SMEs) With Medicinal Products Provided by the PMDA and EMA’. Therapeutic Innovation & Regulatory Science 53, no. 2 (1 March 2019): 193–198.
https://doi.org/10.1177/2168479018771312
Mansnérus, Juli. ‘Encountering Challenges with the EU Regulation on Advance Therapy Medical Products’. European Journal of Health Law 22, no. 5 (2015): 426–461.
https://doi.org/10.1163/15718093-12341369
Pauwels, Kim, Isabelle Huys, Minne Casteels, Kristina Larsson, Caroline Voltz, Karri Penttila, Thomas Morel, and Steven Simoens. ‘Are Products with an Orphan Designation for Oncology Indications Different from Products for Other Rare Indications? A Retrospective Analysis of European Orphan Designations Granted between 2002-2012’. Orphanet Journal of Rare Diseases 12, no. 1 (December 2017): 1–7.
https://doi.org/10.1186/s13023-017-0578-4
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
Putzeist, Michelle, Aukje K. Mantel-Teeuwisse, Christine C. Gispen-De Wied, Arno W. Hoes, and Hubert G. Leufkens. ‘Regulatory Scientific Advice in Drug Development: Does Company Size Make a Difference?’ European Journal of Clinical Pharmacology 67, no. 2 (1 February 2011): 157–164.
https://doi.org/10.1007/s00228-010-0919-x
Reiss, M., I.C. Büttel, and C.K. Schneider. ‘Report from the Committee for Advanced Therapies (CAT) Pitfalls on the way from concept to medical treatment with advanced therapy medicinal products’. Bundesgesundheitsblatt – Gesundheitsforschung – Gesundheitsschutz 54, no. 7 (1 July 2011): 822–830.
https://doi.org/10.1007/s00103-011-1304-3
Salmikangas, Paula, Martina Schuessler-Lenz, Sol Ruiz, Patrick Celis, Ilona Reischl, Margarida Menezes-Ferreira, Egbert Flory, Matthias Renner, and Nicolas Ferry. ‘Marketing Regulatory Oversight of Advanced Therapy Medicinal Products (ATMPs) in Europe: The EMA/CAT Perspective’. In Regulatory Aspects of Gene Therapy and Cell Therapy Products, 103–130. Springer, Cham, 2015.
https://doi.org/10.1007/978-3-319-18618-4_6
Schneider, Christian K., Paula Salmikangas, Bernd Jilma, Bruno Flamion, Lyubina Racheva Todorova, Anna Paphitou, Ivana Haunerova, et al. ‘Challenges with Advanced Therapy Medicinal Products and How to Meet Them’. Nature Reviews Drug Discovery 9, no. 3 (March 2010): 195–201.
https://doi.org/10.1038/nrd3052
Smucr, Bohumil. ‘SME Status – Threat or Opportunity? Opinions’. European State Aid Law Quarterly (ESTAL) 18, no. 4 (2019): 545–548.
Tiedemann, Gudrun, and Sebastian Sethe. ‘Regulatory Frameworks for Cell and Tissue Based Therapies in Europe and the USA’. In Regenerative Medicine, 937–968. Springer, Dordrecht, 2011.
https://doi.org/10.1007/978-90-481-9075-1_39
Van Delm, Kaat. ‘Accelerated Access to Medicinal Products’. European Pharmaceutical Law Review (EPLR) 4, no. 4 (2020): 192–206.
https://doi.org/10.21552/eplr/2020/4/4
