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What Is the Purpose of the Orphan Drug Act?《孤儿药法案》的目的是什么？
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A personal perspective of orphan drug development for rare diseases: A golden opportunity or an unsustainable future?对罕见病孤儿药研发的个人观点：是黄金机遇还是不可持续的未来？
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本文引用的文献

1
Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage.生物标志物定义的常见疾病亚组：《孤儿药法案》覆盖范围的政策与经济影响
PLoS Med. 2017 Jan 3;14(1):e1002190. doi: 10.1371/journal.pmed.1002190. eCollection 2017 Jan.
2
Orphan drug incentives in the pharmacogenomic context: policy responses in the US and Canada.药物基因组学背景下的罕见病药物激励措施：美国和加拿大的政策回应
J Law Biosci. 2015 Apr 19;2(2):263-291. doi: 10.1093/jlb/lsv013. eCollection 2015 Jul.
3
Funding innovation for treatment for rare diseases: adopting a cost-based yardstick approach.为罕见病治疗创新提供资金：采用基于成本的衡量标准方法。
Orphanet J Rare Dis. 2013 Nov 16;8:180. doi: 10.1186/1750-1172-8-180.
4
Orphan drug regulations. Final rule.孤儿药法规。最终规则。
Fed Regist. 2013 Jun 12;78(113):35117-35.
5
Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?支付孤儿药系统费用：是坚持还是妥协？是否到了欧洲的支付方采用新的评估体系来考虑新的罕见病治疗方法的时候了？
Orphanet J Rare Dis. 2012 Sep 26;7:74. doi: 10.1186/1750-1172-7-74.
6
Orphan drug development: an economically viable strategy for biopharma R&D.孤儿药开发：生物制药研发的经济可行策略。
Drug Discov Today. 2012 Jul;17(13-14):660-4. doi: 10.1016/j.drudis.2012.02.005. Epub 2012 Feb 17.
7
An empirical review of major legislation affecting drug development: past experiences, effects, and unintended consequences.影响药物研发的主要立法的实证回顾：过去的经验、影响和意外后果。
Milbank Q. 2011 Sep;89(3):450-502. doi: 10.1111/j.1468-0009.2011.00636.x.
8
Pricing and reimbursement of orphan drugs: the need for more transparency.孤儿药的定价和报销：需要提高透明度。
Orphanet J Rare Dis. 2011 Jun 17;6:42. doi: 10.1186/1750-1172-6-42.
9
Pregnant women deserve better.孕妇应该得到更好的待遇。
Nature. 2010 Jun 10;465(7299):689-90. doi: 10.1038/465689a.
10
The US Orphan Drug Act: rare disease research stimulator or commercial opportunity?美国孤儿药法案：罕见病研究的刺激因素还是商业机会？
Health Policy. 2010 May;95(2-3):216-28. doi: 10.1016/j.healthpol.2009.12.001. Epub 2009 Dec 29.

《孤儿药法案》的目的是什么？

What Is the Purpose of the Orphan Drug Act?

作者信息

Herder Matthew

机构信息

Health Law Institute, Faculties of Medicine and Law, Dalhousie University, Halifax, Nova Scotia, Canada.

出版信息

PLoS Med. 2017 Jan 3;14(1):e1002191. doi: 10.1371/journal.pmed.1002191. eCollection 2017 Jan.

DOI:10.1371/journal.pmed.1002191
PMID:28045908
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5207521/
Abstract

Matthew Herder suggests it may be time to re-examine the purpose of the U.S. Orphan Drug Act.

摘要

马修·赫德认为，或许是时候重新审视美国《孤儿药法案》的目的了。