A bibliographic research was conducted with the expression “Protocol Assistance”, in English and in French on the 25 March 2025, in the following databases: Dalloz, Lexbase, Lexis 360, Lextenso, Navis, Le Doctrinal, Cairn revues, Bibliothèque numérique en droit de la santé, Lamyline Reflex, OpenEdition Journals, Springer Nature, Google Scholar and PubMed.
Please find below the most relevant results of this review:
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
Broz, Daniela Kenzelmann, Gabriele Galliverti, and Anja Buhrer. ‘Scientific Advice Procedures in the EU – an Overview of the Regulatory Background’. Medical Writing 29, no. 3 (September 2020): 36–45.
Elsäßer, Amelie, Jan Regnstrom, Thorsten Vetter, Franz Koenig, Robert James Hemmings, Martina Greco, Marisa Papaluca-Amati, and Martin Posch. ‘Adaptive Clinical Trial Designs for European Marketing Authorization: A Survey of Scientific Advice Letters from the European Medicines Agency’. Trials 15, no. 1 (2 October 2014): 383.
https://doi.org/10.1186/1745-6215-15-383
Gammie, Todd, Christine Y. Lu, and Zaheer Ud-Din Babar. ‘Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries’. PLOS ONE 10, no. 10 (9 October 2015): e0140002.
https://doi.org/10.1371/journal.pone.0140002
Gonzalez-Quevedo, Rosa, Constantinos Ziogas, Ivana Silva, Rosan Vegter, and Anthony Humphreys. ‘Advancing Development of Medicines by Academia and Non-Profit Research Organizations in the European Union’. Nature Reviews Drug Discovery 20, no. 4 (April 2021): 245–246.
https://doi.org/10.1038/d41573-020-00205-x
Hofer, Matthias P., Hanna Hedman, Maria Mavris, Franz Koenig, Thorsten Vetter, Martin Posch, Spiros Vamvakas, Jan Regnstrom, and Stiina Aarum. ‘Marketing Authorisation of Orphan Medicines in Europe from 2000 to 2013’. Drug Discovery Today 23, no. 2 (1 February 2018): 424–433.
https://doi.org/10.1016/j.drudis.2017.10.012
Mariz, Segundo, Tsigkos, Stelios, Fregonese, Laura, Aarum, Stiina, Dehlink, Eleonora, Llinares, Jordi, and Bruno and Sepodes. ‘The Orphan Framework as a New Opportunity: An Expert Opinion’. Expert Opinion on Orphan Drugs 2, no. 11 (1 November 2014): 1181–1186.
https://doi.org/10.1517/21678707.2014.973849
Micallef, Joëlle, and Olivier Blin. ‘Orphan Drug Designation in Europe: A Booster for the Research and Development of Drugs in Rare Diseases’. Therapies, DRUGS AND ORPHAN DISEASES, 75, no. 2 (1 April 2020): 133–139.
https://doi.org/10.1016/j.therap.2020.02.003
Troostenburg, Anne-Ruth van, Giuliana Tabusso, and Enrico Bosone. ‘Scientific Advice and Protocol Assistance by the EMEA’. International Journal of Pharmaceutical Medicine 20, no. 4 (1 August 2006): 227–231.
https://doi.org/10.2165/00124363-200620040-00002
Westermark, Kerstin and The Committee for Orphan Medicinal Products and the European Medicines Agency Scientific Secretariat. ‘European Regulation on Orphan Medicinal Products: 10 Years of Experience and Future Perspectives’. Nature Reviews Drug Discovery 10, no. 5 (May 2011): 341–349.
https://doi.org/10.1038/nrd3445
