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Regulatory and Ethical Issues for Orphan Medicines

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Regulatory and Ethical Issues for Orphan Medicines UNIVERSIDADE DE COIMBRA REGULATORY AND ETHICALISSUESFORORPHANMEDICINES REGULATORY Dissertação de Mestrado em Tecnologias do Medicamento, orientadapeloProfessorDoutorJoãoJoséSousaepela doMedicamento, Dissertação deMestradoemTecnologias Professora Doutora Maria Eugénia Pina e apresentada à Faculdade de Farmácia daUniversidadedeCoimbra deFarmácia Professora DoutoraMariaEugéniaPinaeapresentadaàFaculdade Maria Luísa Borges BatistaBouwman Maria LuísaBorges Junho de2016 Regulatory and Ethical Issues for Orphan Medicines Maria Luísa Borges Batista Bouwman Mestrado em Tecnologias do Medicamento Prof. Doutor João José Sousa Prof.ª Doutora Maria Eugénia Pina Junho de 2016 Universidade de Coimbra When it is obvious that the goals cannot be reached, don´t adjust the goals, adjust the action steps Confucius ii Acknowledgements I would like to thank to my mentors, Professor João José Sousa and Professor Maria Eugénia Pina, for accepting me as their student, knowing very little about my academic past. But also by their ready availability, their incentives and for the contribution with suggestions for improvement. Special thanks to Pedro for the constant support, big patience and for the precious informatic help. To my parents who always supported my dreams, who helped me in my academic travel and giving me strength in many difficult times. Thank you! Agradecimentos Aos meus orientadores, Professor Doutor João José Sousa e Professora Doutora Maria Eugénia Pina, por me terem aceitado como Orientanda, mesmo sabendo muito pouco sobre o meu percurso académico. Mas também pela disponibilidade que sempre demonstraram, pelos incentivos e pela contribuição com sugestões de melhoria. Ao Pedro pelo apoio constante, pela enorme paciência, e pela preciosa ajuda informática. Aos meus pais que apoiaram sempre os meus sonhos, que me ajudaram nesta minha longa caminhada no mundo académico e me deram força nos momentos mais difíceis. Obrigada! iii Contents Acknowledgements .................................................................................................................................... iii Agradecimentos .......................................................................................................................................... iii Acronyms ................................................................................................................................................... viii Graphics Index ........................................................................................................................................... xi Figures Index ............................................................................................................................................... xi Tables Index ............................................................................................................................................... xii Resumo ...................................................................................................................................................... xiii Abstract ..................................................................................................................................................... xiv Introduction ................................................................................................................................................. 1 Presentation of the Project and Goals .................................................................................................. 2 Chapter 1: Orphan Diseases ................................................................................................................ 3 1.1 Rare Diseases.............................................................................................................................. 3 1.1.1. Causes and Examples of Rare Diseases ........................................................................ 4 1.1.2. Epidemiology of Rare Diseases....................................................................................... 8 1.1.2.1. Prevalence Data on Rare Diseases .......................................................................... 10 1.2. Neglected Tropical Diseases (NTDs): overview .............................................................. 11 Chapter 2: Legal Background of Orphan Medicines ..................................................................... 15 2.1 The History of Drug Legislation ........................................................................................... 15 2.2 Food and Drug Administration ............................................................................................. 17 2.2.1 United States of America Definition ........................................................................... 17 2.2.2 Orphan Drug Act ............................................................................................................ 17 2.2.3 How to Apply for Orphan Designation ...................................................................... 18 2.2.4 Economic Assistance and Incentives for Drug Development ................................ 19 2.2.5 Rare Pediatric Disease Priority Review Vouchers ................................................... 21 2.3 European Medicines Agency .................................................................................................. 22 iv 2.3.1 European Definition ........................................................................................................ 23 2.3.2 Orphan Incentives ........................................................................................................... 24 2.3.3 How to Apply for Orphan Designation ...................................................................... 26 2.3.4 Removal of Orphan Designation .................................................................................. 28 2.3.5 Market Authorization and Market Exclusivity ........................................................... 29 2.3.6 Orphan Medicines in Pediatric Population in EU ..................................................... 30 2.3.7 List of orphan medicinal products in Europe with European orphan designation and European marketing authorization: ...................................................................................... 32 2.4 Legislation and the Definition of Orphan Diseases in Different Countries ................ 33 Chapter 3: Patient Network and Advocacy Groups .................................................................... 35 3.1 Umbrella Organizations: EURORDIS and NORD ............................................................ 35 3.1.1 European Rare Diseases Organization (EURORDIS) .............................................. 35 3.1.2 National Organization for Rare Disorders (NORD) .............................................. 37 3.2 European Organizations and Networks ............................................................................. 38 3.2.1 Orphanet ........................................................................................................................... 38 3.2.2 European Platform for Patient Organizations, Science and Industry (EPPOSI) . 40 3.2.3 Patients Network for Medical Research and Health - EGAN ............................... 41 3.3 American Organizations and Networks ............................................................................. 42 3.3.1 Genetic Alliance ............................................................................................................... 42 3.3.2 Rare Disease Clinical Research Network (RDCRN) .............................................. 43 3.4 International Conference for Rare Diseases and Orphan Drugs (ICORD) ............... 44 3.5 Patient Organizations in Portugal ......................................................................................... 45 Chapter 4: Policies and Research Funding ...................................................................................... 46 4.1 Current Model for Financing Drug Development ............................................................ 46 4.1.1 Investors in Drug Development ................................................................................... 48 4.1.2 Status of Investments ...................................................................................................... 49 4.1.3 Ways to Facilitate Drug Development ....................................................................... 49 v 4.1.4 Research Funding in the EU .......................................................................................... 50 4.1.5 Research Funding in the USA ....................................................................................... 51 4.2 Diverse Funding Models ......................................................................................................... 52 4.2.1 Institute for OneWorld Health: A Not-for-profit Pharmaceutical Company ... 53 4.2.2 Cystic Fibrosis Foundation Therapeutics (CFFT): A Virtual Company for Managing Drug Discovery and Development Alliances .......................................................... 54 4.2.3 Genzyme: For-profit Company in the Rare Diseases Arena ................................. 55 4.2.4 Celtic Therapeutics, LLLP: A Private-Equity Model for Addressing Global Health 55 4.3 Strategies for Facilitating Sharing of Research Materials and Data ............................... 56 4.3.1 The Alzheimer’s Disease Neuroimaging Initiative (ADNI): A public-Private Partnership .......................................................................................................................................
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