Citations (14)
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Joon Seok Bang & Jong Hyuk Lee. (2021) The national drug formulary listing process for orphan drugs in South Korea: narrative review focused on pricing and reimbursement pathways. Expert Opinion on Orphan Drugs 9:4, pages 105-112.
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Geoffrey K. Herkes. (2016) Orphan drugs in Australia. Expert Opinion on Orphan Drugs 4:12, pages 1195-1197.
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M Ian Phillips & Andrew B Burns. (2014) The emergence of gene therapy for rare diseases. Expert Opinion on Orphan Drugs 2:11, pages 1197-1209.
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Eline Picavet, David Cassiman & Steven Simoens. (2013) Do ultra-orphan medicinal products warrant ultra-high prices? A review. Orphan Drugs: Research and Reviews 3, pages 23-31.
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John-Paul Michaud, Robin Modi & M Ian Phillips. (2013) Is orphan drug pricing blowing a bubble? The unique situation of orphan drugs and why high prices will likely persist. Expert Opinion on Orphan Drugs 1:9, pages 675-679.
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Peter Norman. (2013) Repurposing as a strategy for orphan drug development, evidence from European approvals. Expert Opinion on Orphan Drugs 1:6, pages 473-480.
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Articles from other publishers (8)
Ryo Okuyama. (2023) Modality diversification and best-in-class small-molecule drugs: Recent trend of orphan drug development. Medicine in Drug Discovery 18, pages 100156.
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Paweł Żelewski, Michał Wojna, Katarzyna Sygit, Elżbieta Cipora, Izabela Gąska, Mateusz Niemiec, Mateusz Kaczmarski, Tomasz Banaś, Beata Karakiewicz, Artur Kotwas, Paulina Zabielska, Olga Partyka, Monika Pajewska, Edyta Krzych-Fałta, Ewa Bandurska, Weronika Ciećko & Aleksandra Czerw. (2022) Comparison of US and EU Prices for Orphan Drugs in the Perspective of the Considered US Orphan Drugs Act Modifications and Discussed Price-Regulation Mechanisms Adjustments in US and European Union. International Journal of Environmental Research and Public Health 19:19, pages 12098.
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Carmine Zoccali, Raymond Vanholder, Carsten A Wagner, Hans-Joachim Anders, Peter J Blankestijn, Annette Bruchfeld, Giovambattista Capasso, Mario Cozzolino, Friedo W Dekker, Danilo Fliser, Denis Fouque, Ron T Gansevoort, Dimitrios Goumenos, Kitty J Jager, Ziad A Massy, Tom A J Oostrom, Ivan Rychlık, Maria Jose Soler, Kate Stevens, Goce Spasovski & Christoph Wanner. (2022) Funding kidney research as a public health priority: challenges and opportunities. Nephrology Dialysis Transplantation 37:1, pages 21-28.
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Proteesh Rana & Shalini Chawla. (2018) Orphan drugs: trends and issues in drug development. Journal of Basic and Clinical Physiology and Pharmacology 29:5, pages 437-446.
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Michael Schlander, Charalabos-Markos Dintsios & Afschin Gandjour. (2018) Budgetary Impact and Cost Drivers of Drugs for Rare and Ultrarare Diseases. Value in Health 21:5, pages 525-531.
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Clemens Stockklausner, Anette Lampert, Georg F. Hoffmann & Markus Ries. (2016) Novel Treatments for Rare Cancers: The U.S. Orphan Drug Act Is Delivering—A Cross-Sectional Analysis. The Oncologist 21:4, pages 487-493.
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Cory Williams. 2014. Orphan Drugs and Rare Diseases. Orphan Drugs and Rare Diseases
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Ashish Kumar Kakkar & Neha Dahiya. (2014) The Evolving Drug Development Landscape: From Blockbusters to Niche Busters in the Orphan Drug Space. Drug Development Research 75:4, pages 231-234.
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