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Orphan drugs revisited.

作者信息

McCabe C, Tsuchiya A, Claxton K, Raftery J

机构信息

Health Economics and Decision Science, School of Health and Related Research, Regent Court, 30 Regent Street, Sheffield S1 4DA.

出版信息

QJM. 2006 May;99(5):341-5; discussion 350-1. doi: 10.1093/qjmed/hcl033. Epub 2006 Feb 27.

DOI:10.1093/qjmed/hcl033
PMID:16504983
Abstract
摘要

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[Orphan drugs: availability, reliability and reimbursement].[罕见病药物：可及性、可靠性与报销]
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Law and research could add up to profitable niche drugs.法律与研究相结合有望催生有利可图的小众药物。
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A fair share for the orphans: ethical guidelines for a fair distribution of resources within the bounds of the 10-year-old European Orphan Drug Regulation.公平分配孤儿资源：在 10 岁的欧洲孤儿药法规范围内公平分配资源的伦理准则。
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Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries.
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