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RECAPTURING THE ORPHAN DRUG ACT: AN ANALYSIS OF PROPOSALS.

• Authors : Trilokekar, Rajdeep

• Source: BYU Journal of Public Law. 2023, Vol. 37 Issue 1, p165-186. 22p.

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Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications.

• Authors : Zhao Z; School of Foreign Languages, China Pharmaceutical University, Nanjing, China.; Institute of Epidemiology and Health Care, University College London, London, UK.

Subjects: Orphan Drug Production* ; Rare Diseases*/Rare Diseases*/Rare Diseases*/drug therapy; Humans

• Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2023 Jul 27; Vol. 18 (1), pp. 220. Date of Electronic Publication: 2023 Jul 27.Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172

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Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma ®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System.

• Authors : Ruggiero, Rosanna^1,2 (AUTHOR) ; Balzano, Nunzia^1,2 (AUTHOR) ; Nicoletti, Maria Maddalena³ (AUTHOR)

• Source: Pharmaceuticals (14248247). Mar2024, Vol. 17 Issue 3, p394. 13p.

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How to START? Four pillars to optimally begin your orphan drug development.

• Authors : Jonker AH; IRDiRC ODDG TF and IRDiRC TSC

Subjects: Orphan Drug Production* ; Rare Diseases*/Rare Diseases*/Rare Diseases*/drug therapy; Humans

• Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2023 Aug 03; Vol. 18 (1), pp. 229. Date of Electronic Publication: 2023 Aug 03.Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172

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A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act.

• Authors : Fermaglich LJ; Office of Orphan Products Development, Office of the Commissioner, US Food and Drug Administration, 10903 New Hampshire Ave, Silver Spring, MD, 20993, USA. .; Miller KL

Subjects: Orphan Drug Production* ; Neoplasms*/Neoplasms*/Neoplasms*/drug therapy; Humans

• Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2023 Jun 23; Vol. 18 (1), pp. 163. Date of Electronic Publication: 2023 Jun 23.Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172

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Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications

• Authors : Zhiyao Zhao; Zhongyang Pei; Anxia Hu

Subjects: Orphan drug development; Rare disease; Incentive policy

• Source: Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-12 (2023)

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The impact of the rare disease and Orphan Drug Act in Taiwan.

• Authors : Ning-Chun Hsiang¹; Weng-Foung Huang²; Churn-Shiouh Gau¹

Subjects: *ORPHAN drug laws; *HEALTH services accessibility; *MEDICAL care costsTAIWAN

• Source: Journal of Food & Drug Analysis. 2021, Vol. 29 Issue 4, p717-725. 9p.

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Cross-national comparative study of orphan drug policies in Saudi Arabia, the United States, and the European Union

• Authors : Bander Balkhi; Asma Almuaither; Saeed Alqahtani

Subjects: Rare disease; Orphan drug; Drug policies

• Source: Saudi Pharmaceutical Journal, Vol 31, Iss 9, Pp 101738- (2023)

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The actual status of drug prices and adjustment factors for drug price calculation: an analysis of ultra-orphan drug development in Japan.

• Authors : Kawakami A; Cooperative Major in Advanced Biomedical Sciences, Joint Graduate School of Tokyo Women's Medical University and Waseda University, 8-1 Kawadacho, Shinjuku-ku, Tokyo, Japan.; Healthcare and Wellness Division, Mitsubishi Research Institute, Inc., 10-3 Nagatacho 2-Chome, Chiyoda-ku, Tokyo, Japan.

Subjects: Drug Costs* ; Drug Development*/Drug Development*/Drug Development*/economics ; Orphan Drug Production*/Orphan Drug Production*/Orphan Drug Production*/economics

• Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2022 Nov 08; Vol. 17 (1), pp. 408. Date of Electronic Publication: 2022 Nov 08.Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172

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Orphan drug policy analysis in China

• Authors : Meilin Liu; Yanqin Lu; Junfeng Li

Subjects: China; rare diseases; orphan drugs

• Source: Frontiers in Pharmacology, Vol 15 (2024)

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