Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries
作者： Todd Gammie , Christine Y. Lu , Zaheer Ud-Din Babar
DOI: 10.1371/JOURNAL.PONE.0140002
关键词: Incentive 、 Public economics 、 Reimbursement 、 Business 、 Pharmacology 、 Orphan Drug Production 、 Marketing authorization 、 Health policy 、 European union 、 Legislation 、 Orphan drug 、 General Biochemistry, Genetics and Molecular Biology 、 General Agricultural and Biological Sciences 、 General Medicine
摘要: 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 synthesised regulations and policies for access to orphan drugs by type and by country. Results Fifty seven articles and 35 countries were included in this review. Six broad categories of regulation and policy instruments were identified: national orphan drug policies, orphan drug designation …
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