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Practice of the Qualification and Recognition for Orphan Drugs in the World and its Inspiration
罕见病研究, 2022 We have analyzed the current status of recognization and qualification of orphan drugs in China and abroad, looking at the aspects of the authority institutions, identification and qualification process, and the number of orphan drugs identified and ...
ZHU Xiaohong +7 more
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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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Expenditure trends of orphan drugs in Serbia: 8-Year analysis of orphan drug market in Serbia [PDF]
Hospital Pharmacology, 2016 Background/Objective: Orphan drugs are medicinal products which treat life-threatening or chronically debilitating diseases which affect small patient populations.
Pejčić Ana V., Iskrov Georgi G.
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Nature, 1992 Diseases lacking in satisfactory therapies, named orphans (over 60% of the known), raise the problem of the availability of new drugs to be discovered and evaluated on toxicological and clinical bases. The estimated cost for the full development of a drug makes it not profitable (and therefore orphan drug) in the case of rare diseases and the typical ...
openaire +4 more sources
Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries. [PDF]
PLoS ONE, 2015 OBJECTIVE:To review existing regulations and policies utilised by countries to enable patient access to orphan drugs. METHODS:A review of the literature (1998 to 2014) was performed to identify relevant, peer-reviewed articles. Using content analysis, we
Todd Gammie +2 more
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罕见病研究, 2022 Comprehensive clinical drug evaluation is an important technical tool for decision-making on drug supply. Traditional evaluation on health technology provides methodological references for comprehensive clinical evalu-ation of drugs.
Multi-disciplinary Team for Rare Diseases, Peking Union Medical College Hospital +1 more
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QJM, 2006 Hughes et al .1 recently discussed arguments for and against giving special funding status to orphan drugs in this journal. They concluded that there should be a uniform policy across Europe, that complete restriction was impractical, and that UK policy should aspire to the values of the EU directive.
McCabe, C. +3 more
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Xiehe Yixue Zazhi, 2022 Comprehensive drug evaluation is an important technical tool for decision-making on drug supply. Traditional health technology of evaluation provides methodological references for comprehensive clinical evalu-ation of drugs.
Multi-disciplinary Team for Rare Diseases, Peking Union Medical College Hospital, National Rare Diseases Committee
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Analysis of patient access to orphan drugs in Turkey
Orphanet Journal of Rare Diseases, 2021 Background Rare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases.
Güvenç Koçkaya +7 more
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Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study
Frontiers in Pharmacology, 2022 Background: Orphan drugs are used for the diagnosis, prevention and treatment of rare diseases that, in the European Union, are defined as disorders affecting no more than 5 persons in 10,000.
Francesca Gorini +23 more
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