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 [PDF]
Orphanet Journal of Rare Diseases, 2023 Background Rare diseases affect more than 30 million Americans. The passage of the Orphan Drug Act (ODA) in the United States in 1983 represented a launching point for a rare disease drug development revolution for these patients.
Lewis J. Fermaglich, Kathleen L. Miller
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Orphan drug development in alpha-1 antitypsin deficiency [PDF]
Scientific Reports, 2022 Alpha-1 antitrypsin deficiency (AATD, OMIM #613490) is a rare metabolic disorder affecting lungs and liver. The purpose of this study is to assess the impact of the US orphan drug act on AATD by providing a quantitative clinical-regulatory insight into ...
Franziska C. Trudzinski +4 more
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Cross-national comparative study of orphan drug policies in Saudi Arabia, the United States, and the European Union [PDF]
Saudi Pharmaceutical Journal, 2023 Background: Rare diseases are chronic, serious, and life-threatening conditions that have not received sufficient attention from drug developers due to their rarity.
Bander Balkhi +2 more
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Long way to go: Progress of orphan drug accessibility in China from 2017 to 2022 [PDF]
Frontiers in Pharmacology, 2023 Introduction: Over 400 million patients worldwide suffer from rare diseases. Access to orphan drugs is, therefore, crucial for this population. China has been actively working on improving orphan drug accessibility in the past decades, especially since ...
Jia Liu +7 more
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Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications [PDF]
Orphanet Journal of Rare Diseases, 2023 Objectives Rare diseases are a global public health issue with a more pressing situation in China. Unfortunately, the relevant research and development in this country are still in its infancy, leading to limited drug accessibility.
Zhiyao Zhao +4 more
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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 [PDF]
Orphanet Journal of Rare Diseases, 2022 Background Extremely high prices facilitate drug development for ultra-rare diseases (ultra-orphan drugs). However, various problems arise in terms of healthcare financing and fairness, and the status of ultra-orphan drug pricing remains ambiguous.
Akihiko Kawakami, Ken Masamune
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Visual Research of Global Orphan Drug from a Bibliometric Perspective [PDF]
Drug Design, Development and TherapyXiaoya Wen,1,* Guangzhi Jin,2,* Chunxing Wu3 1Department of Pharmacy, The Fourth People’s Hospital of GuiYang, Guiyang, Guizhou Province, 550007, People’s Republic of China; 2Department of Hemodialysis Room, The Second People’s Hospital ...
Wen X, Jin G, Wu C
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The Orphan Drug Act at 40: Legislative Triumph and the Challenges of Success. [PDF]
Milbank Q, 2023 Saltonstall P, Ross H, Kim PT.
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Evolving Research and Development Landscape for Rare Diseases: Growing Concerns Over Orphan Drug Lag in Japan. [PDF]
Clin Pharmacol TherEnya K +5 more
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