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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

Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage.

作者信息

Kesselheim Aaron S, Treasure Carolyn L, Joffe Steven

机构信息

Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts, United States of America.

Department of Medical Ethics & Health Policy, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, United States of America.

出版信息

PLoS Med. 2017 Jan 3;14(1):e1002190. doi: 10.1371/journal.pmed.1002190. eCollection 2017 Jan.

DOI:10.1371/journal.pmed.1002190
PMID:28045970
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5207392/
Abstract

Aaron Kesselheim and colleagues examine orphan-designated drugs approved between 2009 and 2015 in the United States.

摘要

亚伦·凯塞尔海姆及其同事研究了2009年至2015年期间在美国获批的孤儿指定药物。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ed09/5207392/0ea113949e89/pmed.1002190.g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ed09/5207392/4bfc66ffaca3/pmed.1002190.g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ed09/5207392/0ea113949e89/pmed.1002190.g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ed09/5207392/4bfc66ffaca3/pmed.1002190.g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ed09/5207392/0ea113949e89/pmed.1002190.g002.jpg

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本文引用的文献

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Orphan drug incentives in the pharmacogenomic context: policy responses in the USA 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. 2016 Jan 7;3(1):158-166. doi: 10.1093/jlb/lsv060. eCollection 2016 Apr.
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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
The High Cost of Prescription Drugs in the United States: Origins and Prospects for Reform.
药物政策与罕见病创新：叙事性综述。
F1000Res. 2023 Nov 13;12:211. doi: 10.12688/f1000research.130809.2. eCollection 2023.
4
Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy.药物研发的特殊 FDA 认定：孤儿药、快速通道、加速批准、优先审评和突破性疗法。
Eur J Health Econ. 2024 Aug;25(6):979-997. doi: 10.1007/s10198-023-01639-x. Epub 2023 Nov 14.
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F1000Res. 2023 Nov 13;12:741. doi: 10.12688/f1000research.130388.1. eCollection 2023.
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BMJ. 2023 May 9;381:e073242. doi: 10.1136/bmj-2022-073242.
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