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]
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]
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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Orphan drugs and the NHS: should we value rarity? [PDF]
Cost effectiveness plays an important part in current decisions about the funding of health technologies. Drugs for rare disease (orphan drugs) are often expensive to produce and, by definition, will benefit only small numbers of patients.
Christopher McCabe+2 more
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Long way to go: Progress of orphan drug accessibility in China from 2017 to 2022 [PDF]
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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Cross-national comparative study of orphan drug policies in Saudi Arabia, the United States, and the European Union [PDF]
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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Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications [PDF]
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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Incentives for orphan drug research and development in the United States [PDF]
Background The Orphan Drug Act (1983) established several incentives to encourage the development of orphan drugs (ODs) to treat rare diseases and conditions.
Rodriguez-Monguio Rosa+3 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]
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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Estimating the clinical cost of drug development for orphan versus non-orphan drugs [PDF]
Background High orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool.
Kavisha Jayasundara+5 more
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Introduction. Drugs used for treatment of rare diseases are known worldwide under the term of orphan drugs because pharmaceutical companies have not been interested in ?adopting? them, that is in investing in research, developing and producing these drugs.
Svetlana Goločorbin-Kon+4 more
openalex +4 more sources