A bibliographic research was conducted with the expression “PRIME”, in English from 2 to 9 April 2025, in the following databases: HeinOnline, Brill revues – Archives ISTEX, Cairn revues, Wiley Online Library, 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:
Antoñanzas, F., C. A. Juárez-Castelló, and R. Rodríguez-Ibeas. ‘EMA Priority Medicines Scheme (PRIME): Will More Paying-for-Performance Agreements Be Needed Due to Immature Data?’ The European Journal of Health Economics 19, no. 7 (2018): 905–907.
https://doi.org/10.1007/s10198-01 7-0944-0
Bloem, Lourens T., Hubert G. M. Leufkens, Sophie E. Berends, Rick A. Vreman, Carla E. M. Hollak, Sonja van Weely, Larissa M. de Lannoy, Peter J. A. Bertens, and Anna M. G. Pasmooij. ‘Stimulating Development of Innovative Medicines in the European Union: Does a New Definition for Unmet Medical Need Add Value?’ Drug Discovery Today 30, no. 1 (1 January 2025): 104251.
https://doi.org/10.1016/j.drudis.2024.104251
Bujar, Magdalena, Neil McAuslane, and Lawrence Liberti. ‘The Qualitative Value of Facilitated Regulatory Pathways in Europe, USA, and Japan: Benefits, Barriers to Utilization, and Suggested Solutions’. Pharmaceutical Medicine 35, no. 2 (1 March 2021): 113–122.
https://doi.org/10.1007/s40290-020-00372-7
Camiel, Adam J. ‘Regulatory Metastasis: Altering the Regulatory and Payer Landscape to Change the Lives of Cancer Patients Notes’. Suffolk Transnational Law Review 40, no. 2 (2017): 279–326.
Chaugule, Purva Dayanand, Priya Changdev Varpe, Ankita Arun Tandulje, Rajeev Singh Raghuvanshi, and Saurabh Srivastava. ‘Expedited Pathway Insights: Unveiling Oncology and Non-Oncology Drug Approvals and Withdrawals of USFDA and EMA’. Critical Reviews in Oncology/Hematology 205 (1 January 2025): 104539.
https://doi.org/10.1016/j.critrevonc.2024.104539
Demirci, Ebru, Grace Omes-Smit, and Alex Zwiers. ‘Clinical Development Time Is Shorter for New Anticancer Drugs Approved via Accelerated Approval in the US or via Conditional Approval in the EU’. Clinical and Translational Science 16, no. 7 (2023): 1127–1133.
https://doi.org/10.1111/cts.13519
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
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 (22 September 2017): 15–19.
https://doi.org/10.1016/j.omtm.2017.08.005
Franco, Pedro, Ritesh Jain, Elizabeth Rosenkrands-Lange, Claudia Hey, and Marén U. Koban. ‘Regulatory Pathways Supporting Expedited Drug Development and Approval in ICH Member Countries’. Therapeutic Innovation & Regulatory Science 57, no. 3 (1 May 2023): 484–514.
https://doi.org/10.1007/s43441-022-00480-3
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
Gonen, Galit. ‘Innovation in Known Drugs – The European Angle Symposium 2017’. Washington Journal of Law, Technology & Arts 12, no. 3 (2017 2016): 253–292.
Hanaizi, Zahra, Sandra Kweder, Shannon Thor, Sonia Ribeiro, and Anabela Marcal. ‘Considering Global Development? Insights from Applications for FDA Breakthrough Therapy and EMA PRIME Designations’. Therapeutic Innovation & Regulatory Science 57, no. 2 (1 March 2023): 321–328.
https://doi.org/10.1007/s43441-022-00475-0
Hawkes, Nigel. ‘European Scheme to Develop Drugs for Diseases with No Treatment Launched’. BMJ: British Medical Journal 352 (8 March 2016).
https://doi.org/10.1136/bmj.i1385
Iglesias-Lopez, Carolina, Mercè Obach, Antonio Vallano, and Antonia Agustí. ‘Comparison of Regulatory Pathways for the Approval of Advanced Therapies in the European Union and the United States’. Cytotherapy 23, no. 3 (1 March 2021): 261–274.
https://doi.org/10.1016/j.jcyt.2020.11.008
Jeong, Hyeokgyo, Sujata Purja, and Eunyoung Kim. ‘Analysis and Comparative Evaluation of Expedited Programs for Gene Therapy Products: Insights from the United States, the European Union, Japan, and South Korea’. Gene Therapy 31, no. 5 (May 2024): 242–254.
https://doi.org/10.1038/s41434-023-00437-7
Jong, Hanna K. de, Sabine M. Hermans, Sophie M. Schuitenmaker, Maya Oli, Mariëtte A. van den Hoven, and Martin P. Grobusch. ‘Factors Associated with Acceleration of Clinical Development for Infectious Diseases: A Cross-Sectional Analysis of 10-Year EMA Registration Data’. The Lancet Regional Health – Europe 43 (1 August 2024): 100983.
https://doi.org/10.1016/j.lanepe.2024.100983
McAuslane, Neil, Lawrence Liberti, and Patricia Connelly. ‘The Confluence of Accelerated Regulatory and Health Technology Assessment Access Pathways’. Clinical Pharmacology & Therapeutics 105, no. 4 (2019): 935–942.
https://doi.org/10.1002/cpt.1315
Muensterman, Elena Tomaselli, Yijia Luo, and Jonathon M. Parker. ‘Breakthrough Therapy, PRIME and Sakigake: A Comparison Between Neuroscience and Oncology in Obtaining Preferred Regulatory Status’. Therapeutic Innovation & Regulatory Science 54, no. 3 (1 May 2020): 658–666.
https://doi.org/10.1007/s43441-019-00100-7
Mullard, Asher. ‘European Regulators Launch “breakthrough” Equivalent Programme’. Nature Reviews Drug Discovery 15, no. 4 (1 April 2016): 223–224.
‘PRIME Time at the EMA’. Nature Reviews Drug Discovery 16, no. 4 (1 April 2017): 226–228.
https://doi.org/10.1038/nrd.2017.57
Nagai, Sumimasa. ‘Flexible and Expedited Regulatory Review Processes for Innovative Medicines and Regenerative Medical Products in the US, the EU, and Japan’. International Journal of Molecular Sciences 20, no. 15 (January 2019): 3801.
https://doi.org/10.3390/ijms20153801
Neez, Emilie, Thomas J. Hwang, Samali Anova Sahoo, and Huseyin Naci. ‘European Medicines Agency’s Priority Medicines Scheme at 2 Years: An Evaluation of Clinical Studies Supporting Eligible Drugs’. Clinical Pharmacology & Therapeutics 107, no. 3 (2020): 541–552.
https://doi.org/10.1002/cpt.1669
Nicotera, Giuseppe, Gianluca Sferrazza, Annalucia Serafino, and Pasquale Pierimarchi. ‘The Iterative Development of Medicines Through the European Medicine Agency’s Adaptive Pathway Approach’. Frontiers in Medicine 6 (27 June 2019).
https://doi.org/10.3389/fmed.2019.00148
Palomo, Gloria M., Tomas Pose-Boirazian, Frauke Naumann-Winter, Enrico Costa, Dinah M. Duarte, Maria E. Kalland, Eva Malikova, et al. ‘The European Landscape for Gene Therapies in Orphan Diseases: 6-Year Experience with the EMA Committee for Orphan Medicinal Products’. Molecular Therapy 31, no. 12 (6 December 2023): 3414–3423.
https://doi.org/10.1016/j.ymthe.2023.09.020
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
Vallano, Antonio, Caridad Pontes, and Antònia Agustí. ‘The Challenges of Access to Innovative Medicines with Limited Evidence in the European Union’. Frontiers in Pharmacology 14 (31 August 2023).
https://doi.org/10.3389/fphar.2023.1215431
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
Wang, Shuhang, Qiuyan Yang, Lan Deng, Qi Lei, Yuqi Yang, Peiwen Ma, Yuxin Men, et al. ‘An Overview of Cancer Drugs Approved through Expedited Approval Programs and Orphan Medicine Designation Globally between 2011 and 2020’. Drug Discovery Today 27, no. 5 (1 May 2022): 1236–1250.
https://doi.org/10.1016/j.drudis.2021.12.021
