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The economics of moonshots: Value in rare disease drug development.

作者信息

Yates Nathan, Hinkel Jennifer

机构信息

ForwardView Consulting, Clintwood, Virginia, USA.

The Data Economics Company, Los Angeles, California, USA.

出版信息

Clin Transl Sci. 2022 Apr;15(4):809-812. doi: 10.1111/cts.13270. Epub 2022 Mar 25.

DOI:10.1111/cts.13270
PMID:35334152
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9010265/
Abstract
摘要

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本文引用的文献

1
Trends In Orphan Drug Spending And Out-Of-Pocket Spending Among US Children, 2013-18.2013 - 2018年美国儿童孤儿药支出及自付费用趋势
Health Aff (Millwood). 2020 Oct;39(10):1806-1811. doi: 10.1377/hlthaff.2020.00595.
2
How many rare diseases are there?有多少种罕见病？
Nat Rev Drug Discov. 2020 Feb;19(2):77-78. doi: 10.1038/d41573-019-00180-y.
3
Variation in US private health plans' coverage of orphan drugs.美国私人健康保险计划对罕见病药物覆盖范围的差异。
Am J Manag Care. 2019 Oct;25(10):508-512.
4
Orphan Cancer Drugs in the Era of Precision Medicine.精准医学时代的孤儿癌症药物。
JAMA Oncol. 2018 Nov 1;4(11):1481-1482. doi: 10.1001/jamaoncol.2018.3498.
5
Evaluating The Impact Of The Orphan Drug Act's Seven-Year Market Exclusivity Period.评估《孤儿药法案》七年市场独占期的影响。
Health Aff (Millwood). 2018 May;37(5):732-737. doi: 10.1377/hlthaff.2017.1179.
6
Rare Disease Terminology and Definitions-A Systematic Global Review: Report of the ISPOR Rare Disease Special Interest Group.罕见病术语与定义——一项全球性系统综述：药物经济学与结果研究国际协会（ISPOR）罕见病特别兴趣小组报告
Value Health. 2015 Sep;18(6):906-14. doi: 10.1016/j.jval.2015.05.008. Epub 2015 Aug 18.
7
Limits on use of health economic assessments for rare diseases.罕见病卫生经济评估的使用限制。
QJM. 2014 Mar;107(3):241-5. doi: 10.1093/qjmed/hcu016. Epub 2014 Jan 22.
8
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.