罕见病药物研发的驱动因素。
Drivers of Orphan Drug Development.
作者信息
Mingorance Ana
机构信息
Dracaena Consulting SL, Madrid 28036, Spain.
出版信息
ACS Med Chem Lett. 2018 Oct 1;9(10):962-964. doi: 10.1021/acsmedchemlett.8b00438. eCollection 2018 Oct 11.
Abstract
Previously neglected by the pharmaceutical industry, rare diseases and orphan drugs are rapidly becoming mainstream. Both market forces and technology enablers are responsible for this migration and combine to create multiple business approaches. This viewpoint discusses the drivers that attract different companies into this booming orphan drug space.
摘要
罕见病和孤儿药此前被制药行业忽视，如今正迅速成为主流。市场力量和技术推动因素共同促成了这一转变，并催生了多种商业模式。本文观点探讨了吸引不同公司进入这一蓬勃发展的孤儿药领域的驱动因素。
相似文献
1
Drivers of Orphan Drug Development.罕见病药物研发的驱动因素。
ACS Med Chem Lett. 2018 Oct 1;9(10):962-964. doi: 10.1021/acsmedchemlett.8b00438. eCollection 2018 Oct 11.
2
Orphan drug legislation: lessons for neglected tropical diseases.罕见病药物立法：被忽视热带病的教训
Int J Health Plann Manage. 2009 Jan-Mar;24(1):27-42. doi: 10.1002/hpm.930.
3
[Early achievements of the Danish pharmaceutical industry--8. Lundbeck].[丹麦制药行业的早期成就——8. 灵北公司]
Theriaca. 2016(43):9-61.
4
Orphan Drugs in Oncology.肿瘤学中的孤儿药
Recent Results Cancer Res. 2019;213:109-142. doi: 10.1007/978-3-030-01207-6_8.
5
Incentives for Starting Small Companies Focused on Rare and Neglected Diseases.创办专注于罕见病和被忽视疾病的小公司的激励措施。
Pharm Res. 2016 Apr;33(4):809-15. doi: 10.1007/s11095-015-1841-9. Epub 2015 Dec 14.
6
Drugs for rare diseases: mixed assessment in Europe.罕见病药物：欧洲的综合评估
Prescrire Int. 2007 Feb;16(87):36-42.
7
The evolving drug development landscape: from blockbusters to niche busters in the orphan drug space.不断演变的药物研发格局：从畅销药到孤儿药领域的小众突破药。
Drug Dev Res. 2014 Jun;75(4):231-4. doi: 10.1002/ddr.21176. Epub 2014 May 14.
8
The Orphan Drug Act: Restoring the Mission to Rare Diseases.《孤儿药法案：恢复针对罕见病的使命》
Am J Clin Oncol. 2016 Apr;39(2):210-3. doi: 10.1097/COC.0000000000000251.
9
Effective Market Exclusivity of New Molecular Entities for Rare and Non-rare Diseases.新分子实体在罕见病和非罕见病方面的有效市场独占权。
Pharmaceut Med. 2020 Feb;34(1):19-29. doi: 10.1007/s40290-019-00317-9.
10
[Historical sketch of modern pharmaceutical science and technology (Part 4). Post World War II 50 years].[现代制药科学技术史略（第4部分）。二战后50年]
Yakushigaku Zasshi. 1995;30(2):75-90.
引用本文的文献
1
The impact of rare diseases on the quality of life in paediatric patients: current status.罕见病对儿科患者生活质量的影响：现状
Front Public Health. 2025 Mar 24;13:1531583. doi: 10.3389/fpubh.2025.1531583. eCollection 2025.
2
The roots of (in)equity in precision medicine: gaps in the discourse.精准医学中（不）公平的根源：话语中的差距。
Per Med. 2024 Jan;21(1):5-9. doi: 10.2217/pme-2023-0097. Epub 2023 Dec 13.
3
Enhancing Equitable Access to Rare Disease Diagnosis and Treatment around the World: A Review of Evidence, Policies, and Challenges.加强全球罕见病诊断和治疗的公平可及性：证据、政策和挑战综述。
Int J Environ Res Public Health. 2023 Mar 8;20(6):4732. doi: 10.3390/ijerph20064732.
4
Adverse Events Related to Off-Label Drugs Using Spontaneous Adverse Event Reporting Systems.使用自发不良事件报告系统报告的与超说明书用药相关的不良事件
Ther Clin Risk Manag. 2021 Aug 21;17:877-887. doi: 10.2147/TCRM.S321789. eCollection 2021.
5
Novel Therapies for Orphan Diseases.罕见病的新型疗法
ACS Med Chem Lett. 2019 Jun 18;10(7):1020-1023. doi: 10.1021/acsmedchemlett.9b00242. eCollection 2019 Jul 11.
本文引用的文献
1
New Therapeutic Uses for Existing Drugs.现有药物的新治疗用途。
Adv Exp Med Biol. 2017;1031:233-247. doi: 10.1007/978-3-319-67144-4_14.
2
Promising gene therapies pose million-dollar conundrum.前景光明的基因疗法带来了百万美元的难题。
Nature. 2016 Jun 16;534(7607):305-6. doi: 10.1038/534305a.
3
The impact of genomics on the future of medicine and health.基因组学对医学与健康未来的影响。
Med J Aust. 2014 Jul 7;201(1):17-20. doi: 10.5694/mja13.10920.
4
The evolving drug development landscape: from blockbusters to niche busters in the orphan drug space.不断演变的药物研发格局：从畅销药到孤儿药领域的小众突破药。
Drug Dev Res. 2014 Jun;75(4):231-4. doi: 10.1002/ddr.21176. Epub 2014 May 14.
5
Orphan drugs: the regulatory environment.孤儿药：监管环境。
Drug Discov Today. 2013 Feb;18(3-4):163-72. doi: 10.1016/j.drudis.2012.08.009. Epub 2012 Sep 5.
6
Diagnosing the decline in pharmaceutical R&D efficiency.诊断制药研发效率下降。
Nat Rev Drug Discov. 2012 Mar 1;11(3):191-200. doi: 10.1038/nrd3681.
7
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.
Zotero 插件