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Tingting Qiu, Yitong Wang, Monique Dabbous, Eve Hanna, Ru Han, Shuyao Liang & Mondher Toumi. (2020) Current state of developing advanced therapies for rare diseases in the European Union. Expert Opinion on Orphan Drugs 8:10, pages 417-429.
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K. E. Young, I. Soussi & M. Toumi. (2017) The perverse impact of external reference pricing (ERP): a comparison of orphan drugs affordability in 12 European countries. A call for policy change. Journal of Market Access & Health Policy 5:1.
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Małgorzata Skweres-Kuchta, Iwona Czerska & Elżbieta Szaruga. (2023) Literature Review on Health Emigration in Rare Diseases—A Machine Learning Perspective. International Journal of Environmental Research and Public Health 20:3, pages 2483.
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Monica Mazzucato, Cinzia Minichiello, Andrea Vianello, Laura Visonà dalla Pozza, Ema Toto & Paola Facchin. (2022) Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study. Frontiers in Pharmacology 13.
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Jong Hyuk Lee. (2021) Pricing and Reimbursement Pathways of New Orphan Drugs in South Korea: A Longitudinal Comparison. Healthcare 9:3, pages 296.
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Carina Schey, Maarten Jacobus Postma, Paul F. M. Krabbe, Olekdandr Topachevskyi, Andrew Volovyk & Mark Connolly. (2020) Assessing the Preferences for Criteria in Multi-Criteria Decision Analysis in Treatments for Rare Diseases. Frontiers in Public Health 8.
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Marcin Czech, Aleksandra Baran-Kooiker, Kagan Atikeler, Maria Demirtshyan, Kamilla Gaitova, Malwina Holownia-Voloskova, Adina Turcu-Stiolica, Coen Kooiker, Oresta Piniazhko, Natella Konstandyan, Olha Zalis'ka & Jolanta Sykut-Cegielska. (2020) A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries. Frontiers in Public Health 7.
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SNEZHANA D. SINYUSHKINA & LEV Y. GARIN. (2020) TO THE QUESTION OF PRICING ON MEDICINES. Herald of the Euro-Asian Law Congress:1, pages 92-99.
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Susana Seca, Diana Miranda, Daniela Cardoso, Bernice Nogueira, Henry J. Greten, António Cabrita & Manuel Alves. (2018) Effectiveness of Acupuncture on Pain, Physical Function and Health-Related Quality of Life in Patients with Rheumatoid Arthritis: A Systematic Review of Quantitative Evidence. Chinese Journal of Integrative Medicine 25:9, pages 704-709.
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Susana SecaMiguel PatrícioSebastian KirchGiovanna FranconiAntónio S. CabritaHenry J. Greten. (2019) Effectiveness of Acupuncture on Pain, Functional Disability, and Quality of Life in Rheumatoid Arthritis of the Hand: Results of a Double-Blind Randomized Clinical Trial. The Journal of Alternative and Complementary Medicine 25:1, pages 86-97.
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Krzysztof Piotr Malinowski, Paweł Kawalec, Wojciech Trabka, Christoph Sowada & Andrzej Pilc. (2018) Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment. Frontiers in Pharmacology 9.
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Andreja Detiček, Igor Locatelli & Mitja Kos. (2018) Patient Access to Medicines for Rare Diseases in European Countries. Value in Health 21:5, pages 553-560.
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Ewout J. N. Groen, Kevin Talbot & Thomas H. Gillingwater. (2018) Advances in therapy for spinal muscular atrophy: promises and challenges. Nature Reviews Neurology 14:4, pages 214-224.
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