A literature review was conducted between 19 and 22 July 2021, with such expressions as orphan drug, orphan medicine, rare disease, orphan regulation, in English and in French, in the following databases: Dalloz, Lextenso, HeinOnline, Lexis Nexis, Navis, Lexbase, Cairn, Westlaw, Brill and Doctrinal Plus. Please find below the most relevant results of this review.
Altavilla, Annagrazia. ‘Introduction: Special Issue on Innovative Medicine and Research: Ethical, Legal and Regulatory Issues’. European Journal of Health Law 27, no. 3 (15 June 2020): 187–93. https://doi.org/10.1163/15718093-BJA10019.
Aulois-Griot, Marine. ‘Les Oubliés de La Santé : À Propos Des Médicaments Orphelins et Des Médicaments Pédiatriques’. RDSS 4 (16 July 2007): 613–22.
Aymé, Ségolène. ‘Histoire des politiques de lutte contre les maladies rares’. Les Tribunes de la santé N° 62, no. 4 (2019): 23–34.
Bache, Gordon and Hervey, Tamara, ‘Incentivising innovation or supporting other interests? European regulation of orphan medicinal products.’ Medical law review 19 no1 (2011): 123 –131.
Benard, Laetitia, Jacqueline Bore, and Eveline Van Keymeulen. ‘Has the Orphan Regulation Met Its Aims’. European Pharmaceutical Law Review (EPLR) 2, no. 4 (2018): 179–92.
Bogaert, Peter. ‘Orphan Medicine Regulation in the EU.’ Global Counseil Supp (Life sciences industry report 2001) (n.d.): 75–81.
Borrás, Xisca. ‘What Has Changed for Orphan Medicinal Products in the UK after Brexit?’ Bristows (blog), 24 May 2021. https://www.bristows.com/news/what-has-changed-for-orphan-medicinal-products-in-the-uk-after-brexit/.
Bouveresse, Aude. ‘Médicaments’. Europe 11, no. 364 (November 2010).
———. ‘Médicaments’. Europe 11, no. 365 (November 2010).
Broes, Stefanie, Sarah Schumacher, Geert Preuveneers, and Isabelle Huys. ‘Intellectual Property Rights and Regulatory Exclusivities in the Pharmaceutical Sector: Challenges and Future Improvements.’ Intellectual Property Quarterly 1 (2016): 19–43.
Cavalier, Gina M. ‘Pushing Parentless Pharmaceuticals: Toward an International Home for Orphan Drugs and a Cure for Zebra Diseases Note’. Law and Policy in International Business 27, no. 2 (1996 1995): 447–76.
Chemtob-Concé, Marie-Catherine. ‘Le Médicament Orphelin : Un Cadre Juridique Incitatif’. Médecine et Droit 81 (1 November 2006): 176–84.
———. ‘Réflexions Sur l’amélioration de La Protection Industrielle Pour Les Médicaments’. Propriété Industrielle 3, no. 6 (March 2010).
Cook, Ros D. ‘Pharmaceuticals: Maintaining a Monopoly.’ BIO-Science Law Review 4, no. 6 (January 2000): 253–57.
De Grove-Valdeyron, Nathalie. ‘Promesses et Faiblesses de La Recherche Stratégique de Souveraineté Européenne Dans Le Domaine Pharmaceutique’. Revue de l’Union Européenne, no. 648 (14 May 2021): 291–98.
Degrassat-Théas, Albane. ‘Les Médicaments Orphelins : Cristallisation Des Tensions Entre Logique Collective et Droit Individuel Sur l’allocation Des Ressources En Santé’. Journal de Droit de La Santé et de l’Assurance Maladie (JDSAM) 4 (1 November 2014): 121–28.
Dennis, Claire. ‘A Comparison of the Pre-Market Orphan Drug Legal Frameworks in the United States and the European Union Notes and Comments’. Wisconsin International Law Journal 35, no. 1 (2018 2017): 138–78.
Dokter, Michiel. ‘The Netherlands: Paediatric Reward for Orphan Drugs.’ Managing Intellectual Property 259 (2016): 99.
Duneau, Michel. ‘Médicaments Orphelins: Une Adoption Laborieuse’. Médecine et Droit 26 (1 September 1997): 7–8.
Faeh, Andrea. ‘A Just Distribution of Health Care in the Case of Orphan Medicinal Products: Aligning the Interests of European Economic Integration and National Welfare Policy.’ European Journal of Social Security 14, no. 1 (2012): 21–40.
Florence, Julia. ‘SPCs and Regulatory Exclusivities.’ Chartered Institute of Patent Agents Journal 46, no. 5 (2017): 16–19.
Galloux, Jean Christophe. ‘Droits Sur Les Créations Nouvelles’. RTD Com., no. 4 (15 December 2000): 900–903.
———. ‘Droits Sur Les Créations Nouvelles’. RTD Com., no. 3 (15 September 2008): 529–36.
Gennet, Éloïse. ‘Introducing “Health Vulnerability”. Towards a Human Right Claim for Innovative Orphan Drugs?’ European Journal of Health Law 27, no. 3 (16 April 2020): 290–307. https://doi.org/10.1163/15718093-BJA10005.
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 (10 November 2017): 541–64. https://doi.org/10.1163/15718093-12341439.
Gibson, Mark. ‘Orphan Drug Legislation: Delivering Incentives to Develop Drugs for Rare Diseases.’ Chartered Institute of Patent Agents Journal 36, no. 5 (2007): 268–72.
Grewal, Jasjote, and Nils Hoppe. ‘Don’t Forget the Orphans.’ European Journal of Health Law 22, no. 2 (2015): 107–11.
Houyez, François. ‘High Price Medicines and Budgets: The Role Patients’ and Consumers’ Organisations Can Play.’ European Journal of Health Law 27, no. 3 (2020): 309–23.
Jolly, Dominique, and Émile Lévy. ‘Le Déficit en G6PD : Arguments épidémiologiques et socio-économiques en faveur de la nécessité d’un dépistage systématique ciblé’. Journal d’économie médicale Volume 28, no. 1 (2010): 19–30.
Jones, Nigel. ‘Regulation and Patenting: European Harmonisation.’ BIO-Science Law Review 99, no. 6 (1998): 203–10.
Kingston, William. ‘How Realistic Are EU Hopes for Innovation?’ European Intellectual Property Review (EIPR) 26, no. 5 (2004): 197–202.
———. ‘Limited Incontestability for Small Firm Patents.’ European Intellectual Property Review (EIPR) 28, no. 9 (2006): 463–68.
Kreeftmeijer-Vegter, Rosan, de Boer, Anthonius, van der Vlugt-Meijer, Roselinda H. et al. ‘The influence of the European paediatric regulation on marketing authorisation of orphan drugs for children.’ Orphanet J Rare Dis 9, 120 (2014). https://doi.org/10.1186/s13023-014-0120-x
Laude, Anne. ‘Médicaments Orphelins’. RDSS 2 (15 June 2000): 371–371.
Le Galès, Catherine. ‘Quel prix pour les médicaments orphelins ?’ Les Tribunes de la santé N° 62, no. 4 (2019): 61–69.
Mahalatchimy, Aurélie, and Taboulet, Florence. ‘L’accès aux médicaments orphelins dans l’Union européenne’, in Le droit de la santé publique dans un contexte transnational, Les Etudes Hospitalières (Duguet Anne-Marie, Ed., Filippi, Isabelle, Carrasco Tristana Co-Eds). Collection « Séminaire d’actualité de droit médical », Bordeaux (2010): 407–22.
Mariz, Segundo, Stelios Tsigkos, Laura Fregonese, Stiina Aarum, Eleonora Dehlink, Jordi Llinares, et Bruno Sepodes. « The Orphan Framework as a New Opportunity: An Expert Opinion ». Expert Opinion on Orphan Drugs 2, no 11 (1 novembre 2014): 1181‑86. https://doi.org/10.1517/21678707.2014.973849.
Mascret, Caroline. ‘La Fixation Du Prix Du Médicament : L’entrée En Scène Du Conseil d’État’. Petites Affiches, no. 88 (2 May 2013): 6.
———. ‘Médicament Orphelin et Procédure de Remboursement En France : L’apport Du Conseil d’État Sur La Question’. Gazette Du Palais, no. 16 (16 January 2010): 35–45.
———. ‘Produits Issus Du Corps Humain, Produits de Santé et Produits Alimentaires ; Médicament Orphelin et Procédure de Remboursement En France : L’apport Du Conseil d’État Sur La Question’. La Gazette Du Palais 15, no. 16 (15 January 2010): 35–39.
Mégerlin, Francis. ‘L’AMM Conditionnelle Issue Du Règlement Communautaire N° 507/2006 et l’urgence de Santé Publique’. RDSS, no. 4 (10 July 2006): 691–703.
Minn, Mari. ‘Development of Orphan Drugs under European Regulatory Incentives and Patent Protection’. European Journal of Health Law 24, no. 3 (2017): 239–63.
Morgan, Gareth. ‘Orphan Drug Status in the EU.’ Pharmaceutical Law Insight 5, no. 4 (2009): 11–12.
Murteira, Susana, Emna El hammi, and Mondher Toumi. ‘Fixing the Price of the Orphan Drug Siklos: The Council of State Takes over the Decision Selected Legislation and Jurisprudence: National’. European Journal of Health Law 21, no. 5 (2014): 505–15.
Novas, Carlos. ‘Orphan Drugs, Patient Activism and Contemporary Healthcare’. Quaderni n° 68, no. 1 (2009): 13.
Papadopoulou, Frantzeska. ‘The Impact of Regulatory Systems in Life Sciences: Emerging Exclusive Rights Growing Outside the Scope of the Traditional Intellectual Property System?’ European Intellectual Property Review (EIPR) 40, no. 5 (2018): 301–10.
Peigné, Jérôme. ‘La Régulation Du Prix Des Médicaments et Le Juge Administratif’. RDSS 6 (30 December 2020): 1039–52.
———. ‘L’affaire Orphacol Ou Le Désaveu Cinglant de La Commission Européenne Pour Avoir Refusé l’AMM à Un Médicament Orphelin’. Journal de Droit de La Santé et de l’Assurance Maladie (JDSAM) 3 (1 October 2013): 72–75.
———. ‘L’intervention Itérative Du Juge Administratif Dans La Fixation Du Prix d’un Médicament Orphelin’. RDSS 4 (10 September 2013): 700–706.
———. ‘Suite de La “Saga Orphacol” : Nouveau Revers Pour La Commission Européenne’. Journal de Droit de La Santé et de l’Assurance Maladie (JDSAM) 3 (1 September 2015): 59–62.
Percy, R. Keith. ‘EC Orphan Drug Regulation.’ Chartered Institute of Patent Agents Journal 27, no. 10 (1998): 753.
Pouppez, Céline. ‘Arrêt “Laboratoires CTRS c. Commission” : Quelle Est La Portée de l’exclusivité Commerciale Des Médicaments Orphelins ?’ Revue Européenne de Droit de La Consommation 1 (1 January 2016): 109–21.
Quinn, Paul, and Paul de Hert. ‘The Patients’ Rights Directive (2011/24/EU) – Providing (Some) Rights to EU Residents Seeking Healthcare in Other Member States.’ Computer Law & Security Review 27, no. 5 (2011): 497–502.
Renaudin, Noël. ‘Le Comité Économique Des Produits de Santé : Dix Ans Après’. RDSS, no. 3 (30 June 2011): 423–29.
Robert, Jacques-Antoine. ‘Prix d’un Médicament Orphelin: Une Décision de Référé Inédite’. Décideurs Juridiques et Financiers 146 (1 February 2013): 24–25.
Robine, Amélie. ‘La Protection Des Données d’autorisation de Mise Sur Le Marché En Droit Communautaire’. RDSS 6 (14 November 2008): 1088–97.
Roset, Sébastien. ‘Circulation Des Marchandises – Médicament’. Europe 3, no. 110 (March 2015).
———. ‘Circulation Des Marchandises – Médicament : Autorisation de Mise Sur Le Marché et Exclusivité Commerciale’. Europe 5, no. 162 (1 May 2016).
———. ‘Circulation Des Marchandises – Médicaments Orphelins -’. Europe 5, no. 182 (May 2018).
Rowland-Rouse, Jacqueline. ‘European Commission – Orphan Drugs.’ BIO-Science Law Review 3, no. 5 (1999): 215.
———. ‘Orphan Drugs Proposal.’ BIO-Science Law Review 00, no. 2 (1999): 77–78.
Sandulli, Aldo. ‘Orphan Drugs for the Treatment of Rare Diseases. A Comparative Public Law Perspective’. Italian Journal of Public Law 11, no. 2 (2019): 476–533.
Sergheraert, Éric, and Anne Soetemont. ‘Actualité Récente de La Procédure de Prorogation Des Certificats Complémentaires de Protection Instituée Par Le « Règlement Pédiatrique »’. Propriété Industrielle 5, no. 10 (May 2009).
Seroc, Samuel. ‘Obligation des autorités nationales, dans l’exercice de leur compétence en matière de remboursement des médicaments, de s’abstenir de prendre des mesures privant un médicament orphelin des bénéfices de son exclusivité commerciale’. Gazette du Palais, no. 9 (3 March 2020): 41.
Syrett, Keith J. ‘Looking after the orphans? Treatments for rare diseases, EU law and the costs of healthcare’. European Law and New Health Technologies. Flear, Mark, Farrell, Maree, Hervey, Tamara and Murphy, Therese (Eds.), Oxford University Press, 2012.
Von Uexkull, Alexa, and Niels Holder. ‘A Heavy Burden.’ Patent World 181 (2006): 9.
Wakeford, Joe. ‘Failure to Spot Rare Disease Did Not Amount to Care Duty Breach.’ Post Magazine, 25 April 2013, 33.
Waller-Davies, Becky. ‘Gene Believer.’ Lawyer 29, no. 20 (2015): 6–7.
Waters, David. ‘Regulatory Data Protection for Medicinal Products for Human Use in the EU.’ Chartered Institute of Patent Agents Journal 32, no. 5 (n.d.): 257–60.
Watson, Kerry. ‘Europe: Orphan Drugs Proposal.’ Patent World 116 (1999): 6–8.
———. ‘European Commission: Orphan Drugs Proposal: Will It Really Promote Research into Rare Diseases?’ BIO-Science Law Review 99, no. 6 (1998): 242–44.
Willoughby, Richard. ‘Are Patents for Orphan Drugs Crucial?’ Managing Intellectual Property 203 Supp (IPFocus Life Sciences), no. 2010 (n.d.): 27–29.