A literature review was conducted with the expression “Compassionate use and advanced therapy medicinal products”, in English and in French between the 10 December 2024 and 7 January 2025, 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
Aartsma-Rus, Annemieke, Alessandra Ferlini, Nathalie Goemans, Anna M.G. Pasmooij, Dominic J. Wells, Katerine Bushby, Elizabeth Vroom, and Pavel Balabanov. ‘Translational and Regulatory Challenges for Exon Skipping Therapies’. Human Gene Therapy 25, no. 10 (October 2014): 885–892. https://doi.org/10.1089/hum.2014.086.
Abbas, Amar, Janis Vella Szijj, Lilian M. Azzopardi, and Anthony Serracino Inglott. ‘Orphan Drug Policies in Different Countries’. Journal of Pharmaceutical Health Services Research 10, no. 3 (2019): 295–302. https://doi.org/10.1111/jphs.12305.
Abdallah, Khadidja, Kathleen Claes, Isabelle Huys, Lennert Follon, Charlotte Calis, and Steven Simoens. ‘Exploring Alternative Financing Models and Early Access Schemes for Orphan Drugs: A Belgian Case Study’. Orphanet Journal of Rare Diseases 17, no. 1 (9 December 2022): 429. https://doi.org/10.1186/s13023-022-02571-8.
Aguiar Gouveia, I., and P. Reis-Pina. ‘The Compassionate Use of Drugs in Palliative Care for Symptom Control and Ethical Justifications for Its Use: A Scoping Review’. Ethics, Medicine and Public Health 31 (1 December 2023): 100950. https://doi.org/10.1016/j.jemep.2023.100950.
Ali, Setti Raïs, Véronique Raimond, Albane Degrassat-Théas, Laurie Rachet Jacquet, Lise Rochaix, Xiaoyan Lu, and Pascal Paubel. ‘Early Access Schemes and Pricing Strategies: A Case Study on Temporary Authorization for Use in France from 1994 to 2016’. Revue Française Des Affaires Sociales, no. 3 (16 November 2018): 69–89. https://doi.org/10.3917/rfas.183.0069.
Artiles-Medina, Alberto, Marina Sáchez-Cuervo, Victoria Gómez-Dos Santos, Emilio J. Sanz-Álvarez, Ana Álvarez-Díaz, and Francisco Javier Burgos-Revilla. ‘Medicines in Exceptional Circumstances for Solid Tumours: Focusing on Evidence, Effectiveness, and Toxicity Profiles’. Farmacia Hospitalaria 46, no. 3 (1 May 2022): 133–145. https://doi.org/10.7399/fh.11854.
Balasubramanian, Gopalakrishnan, Suman Morampudi, Pankdeep Chhabra, and Arun Gowda. ‘An Overview of Compassionate Use Programs in the European Union Member States’. Intractable & Rare Diseases Research, November 2016, 244–254. https://doi.org/10.5582/irdr.2016.01054.
Baran-Kooiker, Aleksandra, Coen Kooiker, and Marcin Czech. ‘Overview of Regulatory Initiatives in the European Union to Stimulate Research and Accelerate Access to Orphan Drugs and Other High Medical Need Products’. ACTA POLONIAE PHARMACEUTICA 76, no. 1 (February 2019): 3–17. https://doi.org/10.32383/appdr/93138.
Berramdane, Abdelkhaleq. ‘Le médicament à usage humain dans le droit de l’Union européenne’. Revue de l’Union européenne | Dalloz, no. 599 (18 June 2016): 363–374.
Bioetica, Comitato Nazionale per la. ‘Single Patient Care and Non-Validated Treatment (The so-Called “Compassionate Use”): Accompanied by a Juridical Note’. Jahrbuch Für Wissenschaft Und Ethik 20, no. 1 (11 July 2016): 385–412. https://doi.org/10.1515/jwiet-2016-0125.
Blanco-Reina, E., A. Munoz-Garcia, M. Cardenas, M. Saldana, E. Rodriguez-Mateos, R. Morillo, J. L. Moreno, et al. ‘Compassionate Use of Medicinal Products: A Multicenter Study’. BASIC & CLINICAL PHARMACOLOGY & TOXICOLOGY 115 (October 2014): 43–44.
Borysowski, Jan, Hans-Jörg Ehni, and Andrzej Górski. ‘Ethics Review in Compassionate Use’. BMC Medicine 15, no. 1 (24 July 2017): 136. https://doi.org/10.1186/s12916-017-0910-9.
Borysowski, Jan, and Andrzej Górski. ‘Compassionate Use of Unauthorized Drugs: Legal Regulations and Ethical Challenges’. European Journal of Internal Medicine 65 (1 July 2019): 12–16. https://doi.org/10.1016/j.ejim.2019.04.008.
Bunnik, Eline M., Nikkie Aarts, and Suzanne van de Vathorst. ‘Little to Lose and No Other Options: Ethical Issues in Efforts to Facilitate Expanded Access to Investigational Drugs’. Health Policy 122, no. 9 (1 September 2018): 977–983. https://doi.org/10.1016/j.healthpol.2018.06.005.
Chu, Céline, Sophie Barbou des Courières, Kevin Fournier, Sophie Kelley, Jacques-Olivier Bay, and Marie Gadeyne. ‘Réforme de l’accès Dérogatoire Aux Médicaments’. Bulletin Du Cancer 109, no. 1 (1 January 2022): 20–22. https://doi.org/10.1016/j.bulcan.2021.11.008.
Cichutek, Klaus, and Ines Krämer. ‘Gene Therapy in Germany and in Europe: Regulatory Issues’. The Quality Assurance Journal 2, no. 3 (1997): 141–152. https://doi.org/10.1002/(SICI)1099-1786(199709)2:3<141::AID-QAJ46>3.0.CO;2-M.
Cooper, Callum J., Mohammadali Khan Mirzaei, and Anders S. Nilsson. ‘Adapting Drug Approval Pathways for Bacteriophage-Based Therapeutics’. Frontiers in Microbiology 7 (3 August 2016). https://doi.org/10.3389/fmicb.2016.01209.
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.
Corthier, M., B. Mézerette, L. Maillant, J. Sales, M. Planel, and E. Sam. ‘PHP368 – THE FRENCH COMPASSIONATE USE OF MEDICINAL PRODUCTS PROGRAM (2012-2017): HEALTH TECHNOLOGY ASSESSMENT AND PRICING IMPLICATIONS’. Value in Health, ISPOR Europe 2018: New Perspectives for Improving 21st Century Health Systems, 21 (1 October 2018): S213. https://doi.org/10.1016/j.jval.2018.09.2867.
Cowling, Tara, Ransi Nayakarathna, Allison L. Wills, Dipti Tankala, Nancy Paul Roc, and Stephane Barakat. ‘Early Access for Innovative Oncology Medicines: A Different Story in Each Nation’. Journal of Medical Economics 26, no. 1 (31 December 2023): 944–953. https://doi.org/10.1080/13696998.2023.2237336.
De Panfilis, Ludovica, Roberto Satolli, and Massimo Costantini. ‘Compassionate Use Programs in Italy: Ethical Guidelines’. BMC Medical Ethics 19, no. 1 (9 March 2018): 22. https://doi.org/10.1186/s12910-018-0263-8.
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): S64–67. https://doi.org/10.1016/j.htct.2021.09.009.
Degrassat-Théas, Albane. ‘La réforme des autorisations temporaires d’utilisation : concilier accès précoce à l’innovation et maîtrise des dépenses de médicaments’. RDSS / Dalloz, no. 4 (27 August 2021): 688–700.
‘Les dispositifs encadrant l’accès aux médicaments innovants à l’hôpital’. RDSS | Dalloz, no. 1 (28 February 2015): 133–143.
Degrassat-Théas, Albane, François Bocquet, Martine Sinègre, Jérôme Peigné, and Pascal Paubel. ‘The “Temporary Recommendations for Use”: A Dual-Purpose Regulatory Framework for off-Label Drug Use in France’. Health Policy 119, no. 11 (1 November 2015): 1399–1405. https://doi.org/10.1016/j.healthpol.2015.09.003.
Denis, Alain, Lut Mergaert, Christel Fostier, Irina Cleemput, and Steven Simoens. ‘A Comparative Study of European Rare Disease and Orphan Drug Markets’. Health Policy 97, no. 2 (1 October 2010): 173–179. https://doi.org/10.1016/j.healthpol.2010.05.017.
Deplanque, Dominique, Christine Fetro, Antoine Ferry, Philippe Lechat, Terence Beghyn, Claude Bernard, Antoine Bernasconi, et al. ‘Drug Repurposing: From the Discovery of a Useful Pharmacological Effect to Making the Treatment Available to the Patient’. Therapies, GIENS WORKSHOPS (29-30 June 2022), 78, no. 1 (1 January 2023): 10–18. https://doi.org/10.1016/j.therap.2022.11.009.
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.
Diab-Cáceres, Layla, Rosa María Girón-Moreno, María Teresa Pastor-Sanz, Esther Quintana-Gallego, Isabel Delgado-Pecellín, Marina Blanco-Aparicio, Luis Maiz, et al. ‘Compassionate Use of Lumacaftor/Ivacaftor in Cystic Fibrosis: Spanish Experience’. Archivos de Bronconeumología (English Edition) 54, no. 12 (1 December 2018): 614–618. https://doi.org/10.1016/j.arbr.2018.05.020.
Dodds-Smith, Ian, and Ewan Townsend. ‘The Supply of Unlicensed Medicines for Individual Patient Use’. In The Textbook of Pharmaceutical Medicine, 610–631. John Wiley & Sons, Ltd, 2013. https://doi.org/10.1002/9781118532331.ch31.
Dominici, Massimo, Karen M. Nichols, Aaron D. Levine, John EJ Rasko, Miguel Forte, Lynn O’Donnell, Mickey BC Koh, Catherine M. Bollard, and Daniel J. Weiss. ‘Science, Ethics and Communication Remain Essential for the Success of Cell-Based Therapies’. Brain Circulation 2, no. 3 (September 2016): 146. https://doi.org/10.4103/2394-8108.192525.
Dory, A., and B. Gourieux. ‘Les nouveaux dispositifs d’accès précoce aux innovations thérapeutiques: New early access program for drug innovation’. Revue des Maladies Respiratoires Actualités, Cours du Groupe d’Oncologie thoracique de Langue Française – GOLF 2023, 15, no. 2, Supplement 1 (1 October 2023): 2S130–139. https://doi.org/10.1016/S1877-1203(23)00146-5.
‘EBE and EuropaBio White Paper “Towards an Optimal Orphan Medicinal Products (OMP) Framework in Europe” [Excerpts] Texts’. Biotechnology Law Report 24, no. 3 (2005): 370–381.
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.
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.
Feltmate, Karen, Peter M. Janiszewski, Sheena Gingerich, and Michael Cloutier. ‘Delayed Access to Treatments for Rare Diseases: Who’s to Blame?’ Respirology 20, no. 3 (2015): 361–369. https://doi.org/10.1111/resp.12498.
Ferry, Tristan, Camille Kolenda, Thomas Briot, Aubin Souche, Sébastien Lustig, Jérôme Josse, Cécile Batailler, et al. ‘Past and Future of Phage Therapy and Phage-Derived Proteins in Patients with Bone and Joint Infection’. Viruses 13, no. 12 (December 2021): 2414. https://doi.org/10.3390/v13122414.
Fontanals, Sandra, Anna Esteve, Andrea González, Cristina Ibáñez, Javier Martínez, Ricard Mesía, and Ana Clopés. ‘Real-World Treatment Outcomes of Medicines Used in Special Situations (off-Label and Compassionate Use) in Oncology and Hematology: A Retrospective Study from a Comprehensive Cancer Institution’. Cancer Medicine 12, no. 16 (2023): 17112–17125. https://doi.org/10.1002/cam4.6360.
Fountzilas, Elena, Rabih Said, and Apostolia M. Tsimberidou. ‘Expanded Access to Investigational Drugs: Balancing Patient Safety with Potential Therapeutic Benefits’. Expert Opinion on Investigational Drugs 27, no. 2 (1 February 2018): 155–162. https://doi.org/10.1080/13543784.2018.1430137.
Frati, Paola, Giacomo Frati, Matteo Gulino, Gianluca Montanari Vergallo, Alessandro Di Luca, and Vittorio Fineschi. ‘Stem Cell Therapy: From Evidence-Based Medicine to Emotion-Based Medicine? The Long Italian Way for a Scientific Regulation’. Stem Cell Research & Therapy 4, no. 5 (11 October 2013): 122. https://doi.org/10.1186/scrt333.
Frenken, Gregor, and Hannah Lüttge. ‘Regulation of Genome Editing in Human IPS Cells: Germany’. In Regulation of Genome Editing in Human IPS Cells: A Comparative Legal Analysis of National Regulatory Frameworks for IPSC-Based Cell/Gene Therapies, edited by Hans-Georg Dederer and Gregor Frenken, 29–81. Springer International Publishing, 2022. https://doi.org/10.1007/978-3-030-93023-3_3.
Frutos Pérez-Surio, A., and J. R. Lozano Ortíz. ‘The Use of Medicines in Exceptional Circumstances in Spain: Adding Perspective to Early Access’. Drugs & Therapy Perspectives 35, no. 2 (1 February 2019): 86–92. https://doi.org/10.1007/s40267-018-0590-3.
Gardner, John, Ruchi Higham, Alex Faulkner, and Andrew Webster. ‘Promissory Identities: Sociotechnical Representations & Innovation in Regenerative Medicine’. Social Science & Medicine 174 (1 February 2017): 70–78. https://doi.org/10.1016/j.socscimed.2016.12.018.
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 (22 July 2023): 1–19. https://doi.org/10.3390/ijms241411803.
González-Morcillo, Gonzalo, Beatriz Calderón-Hernanz, and Juan Manuel Rodríguez-Camacho. ‘Cost-Effectiveness of Drug Therapy Prescribed in Special Situations at Hospital’. Farmacia Hospitalaria 44, no. 6 (1 November 2020): 272–278. https://doi.org/10.7399/fh.11451.
Górski, Andrzej, Ryszard Międzybrodzki, Beata Weber-Dąbrowska, Wojciech Fortuna, Sławomir Letkiewicz, Paweł Rogóż, Ewa Jończyk-Matysiak, Krystyna Dąbrowska, Joanna Majewska, and Jan Borysowski. ‘Phage Therapy: Combating Infections with Potential for Evolving from Merely a Treatment for Complications to Targeting Diseases’. Frontiers in Microbiology 7 (26 September 2016). https://doi.org/10.3389/fmicb.2016.01515.
Hunter, Philip. ‘Advanced Therapies Push Regulatory Boundaries’. EMBO Reports 18, no. 12 (December 2017): 2101–2104. https://doi.org/10.15252/embr.201745345.
Hyry, Hanna I., Jeremy Manuel, Timothy M. Cox, and Jonathan C. P. Roos. ‘Compassionate Use of Orphan Drugs’. Orphanet Journal of Rare Diseases 10, no. 1 (21 August 2015): 100. https://doi.org/10.1186/s13023-015-0306-x.
‘III. Dokumentation’. Jahrbuch Für Wissenschaft Und Ethik 8, no. 1 (31 December 2003): 383–348. https://doi.org/10.1515/9783110243963.383.
Iudicello, Antonella, Lucia Alberghini, Giulia Benini, and Paola Mosconi. ‘Expanded Access Programme: Looking for a Common Definition’. Trials 17, no. 1 (12 January 2016): 21. https://doi.org/10.1186/s13063-015-1108-0.
Jacquet, Emmanuelle, Ghania Kerouani-Lafaye, Francoise Grude, Sergio Goncalves, Annie Lorence, Florence Turcry, Liora Brunel, et al. ‘Comparative Study on Anticancer Drug Access Times between FDA, EMA and the French Temporary Authorisation for Use Program over 13 Years’. European Journal of Cancer 149 (1 May 2021): 82–90. https://doi.org/10.1016/j.ejca.2021.03.008.
Kacetl, Jaroslav, Petra Marešová, Raihan Maskuriy, Raihan, and Ali Selamat. ‘Ethical Questions Linked to Rare Diseases and Orphan Drugs – A Systematic Review’. Risk Management and Healthcare Policy, no. 13 (2020): 2125–2148.
Korde, Aruna, Marianne Patt, Svetlana V. Selivanova, Andrew M. Scott, Rolf Hesselmann, Oliver Kiss, Natesan Ramamoorthy, et al. ‘Position Paper to Facilitate Patient Access to Radiopharmaceuticals: Considerations for a Suitable Pharmaceutical Regulatory Framework’. EJNMMI Radiopharmacy and Chemistry 9, no. 1 (2 January 2024): 2. https://doi.org/10.1186/s41181-023-00230-2.
Krendyukov, Andriy. ‘Early Access Provision for Innovative Medicinal Products in Oncology: Challenges and Opportunities’. Frontiers in Oncology 10 (2 September 2020). https://doi.org/10.3389/fonc.2020.01604.
Kuehn, Lauren. ‘Do Desperate Times Really Call for Desperate Measures? The Ethical Dilemma Behind the Regulation and Use of Experimental Drugs’. Notre Dame Journal of International & Comparative Law 7, no. 2 (3 May 2017). https://scholarship.law.nd.edu/ndjicl/vol7/iss2/8.
Lamb, Louise, and Paolo M. Biffignandi. ‘Review 2001: The Future of European Pharmaceutical Legislation’. Drug Information Journal : DIJ / Drug Information Association 36, no. 4 (1 October 2002): 899–907. https://doi.org/10.1177/009286150203600419.
Lançon, Christophe, Mondher Toumi, Christophe Sapin, and Karina Hansen. ‘The Sertindole Safety Survey: A Retrospective Analysis under a Named Patient Use Programme in Europe’. BMC Psychiatry 8, no. 1 (18 July 2008): 57. https://doi.org/10.1186/1471-244X-8-57.
Löblová, Olga, Marcell Csanádi, Piotr Ozierański, Zoltán Kaló, Lawrence King, and Martin McKee. ‘Alternative Access Schemes for Pharmaceuticals in Europe: Towards an Emerging Typology’. Health Policy 123, no. 7 (1 July 2019): 630–634. https://doi.org/10.1016/j.healthpol.2019.05.012.
‘Patterns of Alternative Access: Unpacking the Slovak Extraordinary Drug Reimbursement Regime 2012-2016’. Health Policy 123, no. 8 (1 August 2019): 713–720. https://doi.org/10.1016/j.healthpol.2019.05.021.
Lutsyk, Karyna, Tristan Gicquel, Lucas Cortial, Sylvain Forget, Serge Braun, Pierre-Olivier Boyer, Vincent Laugel, and Olivier Blin. ‘Does Gene Therapies Clinical Research in Rare Diseases Reflects the Competitivity of the Country: Example of France’. Therapies 79, no. 5 (1 September 2024): 505–518. https://doi.org/10.1016/j.therap.2024.01.007.
Mahalatchimy, Aurélie. ‘Pour une stratégie de l’Union européenne dans le domaine de l’innovation en santé’. Revue de l’Union européenne | Dalloz, no. 6 (27 August 2021): 22–29.
———. ‘Regulation of Genome Editing in Human IPS Cells: France’. In Regulation of Genome Editing in Human IPS Cells: A Comparative Legal Analysis of National Regulatory Frameworks for IPSC-Based Cell/Gene Therapies, edited by Hans-Georg Dederer and Gregor Frenken, 83–106. Springer International Publishing, 2022. https://doi.org/10.1007/978-3-030-93023-3_4.
Mahalatchimy, Aurélie, and Nathalie De Grove-Valdeyron. ‘Les essais cliniques de médicaments de thérapie innovante : quel avenir pour la réglementation européenne ?’ Journal international de bioéthique et d’éthique des sciences 29, no. 2 (27 July 2018): 35–51. https://doi.org/10.3917/jibes.292.0035.
Mariz, Segundo, Stelios Tsigkos, Laura Fregonese, Stiina Aarum, Eleonora Dehlink, Jordi Llinares, and Bruno 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.
Martin, J Elliott, Colin Butler, and Aikaterini Varanou-Jenkins. ‘Tracheal Replacement Therapy with a Stem Cell-Seeded Graft: Lessons from Compassionate Use Application of a GMP-Compliant Tissue-Engineered Medicine’. Stem Cells Translational Medicine 6, no. 6 (1 June 2017): 1458–1464. https://doi.org/10.1002/sctm.16-0443.
Melchior, Rosanna. ‘A Time for COMPASSION’. actmagazine.com, September 2007. https://www.proquest.com/openview/fa1b1e74e894929ea3078752ae9640b3/1?pq-origsite=gscholar&cbl=44052.
Mills, Mackenzie, and Panos Kanavos. ‘How Do HTA Agencies Perceive Conditional Approval of Medicines? Evidence from England, Scotland, France and Canada’. Health Policy 126, no. 11 (1 November 2022): 1130–1143. https://doi.org/10.1016/j.healthpol.2022.08.005.
Minghetti, Paola, Iolanda Palmieri, and Francesca Selmin. ‘When Authorized Medicinal Products Are Not Available: Possible Alternatives to Meet Legitimate Expectations of Patients’. Journal of Pharmaceutical Health Services Research 1, no. 3 (2010): 107–112. https://doi.org/10.1111/j.1759-8893.2010.00018.x.
Moine-Dupuis, Isabelle, and Erika Lietzan. ‘Lièvre ou tortue ? Les accès anticipés au médicament à l’épreuve du dilemme entre précaution et « droit à l’espoir » des patients’. RDSS | Dalloz, no. 2 (30 April 2021): 289–301.
Morpurgo, Marco de. ‘Italy Reforms Clinical Trial Rules Reports: Italy’. European Pharmaceutical Law Review (EPLR) 2, no. 1 (2018): 32–36. https://doi.org/10.21552/eplr/2018/1/7.
Muñoz, M., J. A. García-Erce, I. Villar, and D. Thomas. ‘Blood Conservation Strategies in Major Orthopaedic Surgery: Efficacy, Safety and European Regulations’. Vox Sanguinis 96, no. 1 (2009): 1–13. https://doi.org/10.1111/j.1423-0410.2008.01108.x.
Naumann-Winter, Frauke, Franziska Wolter, Ulrike Hermes, Eva Malikova, Nils Lilienthal, Tania Meier, Maria Elisabeth Kalland, and Armando Magrelli. ‘Licensing of Orphan Medicinal Products—Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021’. Frontiers in Pharmacology 13 (11 August 2022). https://doi.org/10.3389/fphar.2022.920336.
Pace, Jessica, Narcyz Ghinea, Ian Kerridge, and Wendy Lipworth. ‘An Ethical Framework for the Creation, Governance and Evaluation of Accelerated Access Programs’. Health Policy 122, no. 9 (1 September 2018): 984–990. https://doi.org/10.1016/j.healthpol.2018.07.014.
Patey, Olivier, Shawna McCallin, Hubert Mazure, Max Liddle, Anthony Smithyman, and Alain Dublanchet. ‘Clinical Indications and Compassionate Use of Phage Therapy: Personal Experience and Literature Review with a Focus on Osteoarticular Infections’. Viruses 11, no. 1 (January 2019): 18. https://doi.org/10.3390/v11010018.
PERIN, LEONARDO. ‘Reliability of Real-world data: a review and meta-analysis to evaluate the consistency of Real-world data collection from Compassionate Use programs and corresponding Registration trials’. UNIVERSITA’ DEGLI STUDI DI PADOVA, 2024. https://thesis.unipd.it/handle/20.500.12608/68649.
Picavet, Eline, David Cassiman, and Steven Simoens. ‘Evaluating and Improving Orphan Drug Regulations in Europe: A Delphi Policy Study’. Health Policy 108, no. 1 (1 November 2012): 1–9. https://doi.org/10.1016/j.healthpol.2012.08.023.
Pinxten, Wim, Herman Nys, and Kris Dierickx. ‘Access to Investigational Medicinal Products for Minors in Europe: Ethical and Regulatory Issues in Negotiating Children’s Access to Investigational Medicines’. Journal of Medical Ethics 36, no. 12 (2010): 791–794. https://doi.org/10.1136/jme.2010.036442 791.
Poulos, Jordan. ‘The Limited Application of Stem Cells in Medicine: A Review’. Stem Cell Research & Therapy 9, no. 1 (2 January 2018): 1. https://doi.org/10.1186/s13287-017-0735-7.
Rahbari, Mussa, and Nuh N. Rahbari. ‘Compassionate Use of Medicinal Products in Europe: Current Status and Perspectives’. Bulletin of the THE WORLD HEALTH ORGANIZATION 89, no. 3 (March 2011): 163–163. https://doi.org/10.2471/BLT.10.085712.
Raposo, Vera Lucia. ‘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 1, no. 29 (March 2022): 504–520. https://doi.org/10.1163/15718093-bja10083.
Raposo, Vera Lucia, and Li Du. ‘Stem Cell Based Products in Europe and in China: Where Are We and Where Should We Go?’ European Pharmaceutical Law Review (EPLR) 4, no. 3 (2020): 161–171. https://doi.org/10.21 552/epIr/2020/3/6.
Raus, Kasper. ‘An Analysis of Common Ethical Justifications for Compassionate Use Programs for Experimental Drugs’. BMC Medical Ethics 17, no. 1 (18 October 2016): 60. https://doi.org/10.1186/s12910-016-0145-x.
Reichart, Bruno, David K C Cooper, Matthias Längin, Ralf R Tönjes, Richard N Pierson III, and Eckhard Wolf. ‘Cardiac Xenotransplantation: From Concept to Clinic’. Cardiovascular Research 118, no. 18 (7 December 2022): 3499–3516. https://doi.org/10.1093/cvr/cvac180.
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.
Rosenberg, Noa, Sibren van den Berg, Nina N. Stolwijk, Bart A. W. Jacobs, Hendrika C. Post, Anna M. G. Pasmooij, Saco J. de Visser, and Carla E. M. Hollak. ‘Access to Medicines for Rare Diseases: A European Regulatory Roadmap for Academia’. Frontiers in Pharmacology 14 (28 February 2023). https://doi.org/10.3389/fphar.2023.1142351.
Saint-Raymond, A, J Sato, Y Kishioka, T Teixeira, C Hasslboeck, and SL Kweder. ‘Remdesivir Emergency Approvals: A Comparison of the U.S., Japanese, and EU Systems’. Expert Review of Clinical Pharmacology 13, no. 10 (2 October 2020): 1095–1101. https://doi.org/10.1080/17512433.2020.1821650.
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