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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 [PDF]
International Journal of Environmental Research and Public Health, 2022 Olga Partyka +2 more
exaly +2 more sources
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
doaj +1 more source
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
doaj +1 more source
Medicinski pregled, 2013 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 Golocorbin-Kon +4 more
+6 more sources
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.
doaj +1 more source
Clinical and Translational Science, 2022 Clinical pharmacology is an integral discipline supporting the development, regulatory evaluation, and clinical use of drugs for the treatment of both common and rare diseases.
Julie Hsieh +4 more
doaj +1 more source
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
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
doaj +1 more source
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
doaj +1 more source
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
doaj +1 more source