• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利：CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

文献检索

告别复杂PubMed语法，用中文像聊天一样搜索，搜遍4000万医学文献。AI智能推荐，让科研检索更轻松。

立即免费搜索

文件翻译

保留排版，准确专业，支持PDF/Word/PPT等文件格式，支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述，25分钟生成高质量综述，智能提取关键信息，辅助科研写作。

立即免费体验

Editorial: The use of real world data for regulatory purposes in the rare diseases setting.

作者信息

Giannuzzi Viviana, Stoyanova-Beninska Violeta, Hivert Virginie

机构信息

Research Department, Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus, Valenzano, Italy.

Medicines Evaluation Board, Utrecht, Netherlands.

出版信息

Front Pharmacol. 2022 Nov 24;13:1089033. doi: 10.3389/fphar.2022.1089033. eCollection 2022.

DOI:10.3389/fphar.2022.1089033
PMID:36506540
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9730868/
Abstract
摘要

相似文献

1
Editorial: The use of real world data for regulatory purposes in the rare diseases setting.社论：在罕见病领域将真实世界数据用于监管目的
Front Pharmacol. 2022 Nov 24;13:1089033. doi: 10.3389/fphar.2022.1089033. eCollection 2022.
2
Licensing of Orphan Medicinal Products-Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021.孤儿药用产品许可——2019年至2021年在欧洲药品管理局完成的上市许可申请中关于疗效方面真实世界数据及其他外部数据的使用
Front Pharmacol. 2022 Aug 11;13:920336. doi: 10.3389/fphar.2022.920336. eCollection 2022.
3
[The role of real-world evidence for the study of rare diseases epidemiology and the post-marketing evaluation of orphan drugs.].[真实世界证据在罕见病流行病学研究及孤儿药上市后评估中的作用。]
Recenti Prog Med. 2022 Jul-Aug;113(7):425-429. doi: 10.1701/3850.38336.
4
Role of healthcare databases and registries for surveillance of orphan drugs in the real-world setting: the Italian case study.真实环境下的医疗保健数据库和注册中心在孤儿药监测中的作用：意大利的案例研究。
Expert Opin Drug Saf. 2019 Jun;18(6):497-509. doi: 10.1080/14740338.2019.1614165. Epub 2019 May 22.
5
The effect of adding real-world evidence to regulatory submissions on the breadth of population indicated for rare disease medicine treatment by the European Medicines Agency.在监管申报中加入真实世界证据对欧洲药品管理局指定的罕见病药物治疗适用人群广度的影响。
J Pharm Policy Pract. 2022 May 4;15(1):36. doi: 10.1186/s40545-022-00433-z.
6
Orphan Medicines for Pediatric Use: A Focus on the European Union.儿科孤儿药：以欧盟为例。
Clin Ther. 2019 Dec;41(12):2630-2642. doi: 10.1016/j.clinthera.2019.10.006. Epub 2019 Nov 5.
7
Access and pricing of medicines for patients with rare diseases in the European Union: an industry perspective.欧盟罕见病患者的药物可及性和定价：行业视角。
Expert Rev Pharmacoecon Outcomes Res. 2022 Apr;22(3):381-389. doi: 10.1080/14737167.2022.2020105. Epub 2022 Jan 7.
8
Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study.罕见病（RD）患者中孤儿药（OMPs）的真实世界使用情况：一项基于人群的注册研究。
Front Pharmacol. 2022 Sep 30;13:940010. doi: 10.3389/fphar.2022.940010. eCollection 2022.
9
Uptake of orphan drugs in the WHO essential medicines lists.罕见病药物在世界卫生组织基本药物清单中的应用情况。
Bull World Health Organ. 2024 Jan 1;102(1):22-31. doi: 10.2471/BLT.23.289731. Epub 2023 Oct 31.
10
Market access for medicines treating rare diseases: Association between specialised processes for orphan medicines and funding recommendations.罕见病药物的市场准入：孤儿药专门审批程序与资金推荐之间的关联。
Soc Sci Med. 2022 Aug;306:115119. doi: 10.1016/j.socscimed.2022.115119. Epub 2022 Jun 8.

引用本文的文献

1
The Potential to Leverage Real-World Data for Pediatric Clinical Trials: A Proof-of-Concept Study.利用真实世界数据进行儿科临床试验的潜力：一项概念验证研究。
J Med Internet Res. 2025 May 30;27:e72573. doi: 10.2196/72573.
2
Utility of Clustering in Mortality Risk Stratification in Pulmonary Hypertension.聚类分析在肺动脉高压死亡率风险分层中的应用
Bioengineering (Basel). 2025 Apr 11;12(4):408. doi: 10.3390/bioengineering12040408.
3
Sharing Data and Transferring Samples Within Pediatric Clinical Studies: How to Overcome Challenges and Make Them a Science Opportunity.儿科临床研究中的数据共享与样本转移：如何克服挑战并将其转化为科学机遇。
Healthcare (Basel). 2024 Dec 6;12(23):2473. doi: 10.3390/healthcare12232473.
4
The creation of an adaptable informed consent form for research purposes to overcome national and institutional bottlenecks in ethics review: experience from rare disease registries.创建一份适用于研究目的的知情同意书，以克服伦理审查中的国家和机构瓶颈：来自罕见病登记处的经验。
Front Med (Lausanne). 2024 Apr 17;11:1384026. doi: 10.3389/fmed.2024.1384026. eCollection 2024.
5
Innovative research methodologies in the EU regulatory framework: an analysis of EMA qualification procedures from a pediatric perspective.欧盟监管框架中的创新研究方法：从儿科角度分析欧洲药品管理局的资格认定程序。
Front Med (Lausanne). 2024 Mar 28;11:1369547. doi: 10.3389/fmed.2024.1369547. eCollection 2024.
6
Minimum data set for treatment effectiveness in pyoderma gangrenosum (MIDSTEP): an international protocol of an e-Delphi study to develop a clinical physician-driven treatment effectiveness registry on behalf of the UPGRADE initiative.坏疽性脓皮病治疗效果最小数据集（MIDSTEP）：一项国际电子德尔菲研究协议，旨在代表 UPGRADE 倡议开发一个由临床医生驱动的治疗效果登记处。
Arch Dermatol Res. 2023 Dec;315(10):2913-2919. doi: 10.1007/s00403-023-02729-9. Epub 2023 Sep 27.

本文引用的文献

1
Creating standards for Canadian health data protection during health emergency - An analysis of privacy regulations and laws.制定卫生紧急情况期间加拿大健康数据保护标准——隐私法规与法律分析
Heliyon. 2022 May 21;8(5):e09458. doi: 10.1016/j.heliyon.2022.e09458. eCollection 2022 May.
2
Enablers and barriers to the secondary use of health data in Europe: general data protection regulation perspective.欧洲健康数据二次使用的推动因素与障碍：从《通用数据保护条例》角度分析
Arch Public Health. 2022 Apr 9;80(1):115. doi: 10.1186/s13690-022-00866-7.
3
Stride Velocity 95th Centile: Insights into Gaining Regulatory Qualification of the First Wearable-Derived Digital Endpoint for use in Duchenne Muscular Dystrophy Trials.步速第 95 百分位：在杜氏肌营养不良症试验中获得首款可穿戴衍生数字终点的监管资格的见解。
J Neuromuscul Dis. 2022;9(2):335-346. doi: 10.3233/JND-210743.
4
First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases.杜氏肌营养不良症新型数字终点的首次监管认定：多利益相关方对神经肌肉疾病患者及药物研发影响的观点
Digit Biomark. 2021 Aug 5;5(2):183-190. doi: 10.1159/000517411. eCollection 2021 May-Aug.
5
Marketing Authorization Applications Made to the European Medicines Agency in 2018-2019: What was the Contribution of Real-World Evidence?2018-2019 年向欧洲药品管理局提交的营销授权申请：真实世界证据的贡献是什么？
Clin Pharmacol Ther. 2022 Jan;111(1):90-97. doi: 10.1002/cpt.2461. Epub 2021 Nov 13.
6
Privacy protection of medical data in social network.社交网络中医疗数据的隐私保护。
BMC Med Inform Decis Mak. 2021 Oct 18;21(Suppl 1):286. doi: 10.1186/s12911-021-01645-0.
7
Privacy Protection and Secondary Use of Health Data: Strategies and Methods.隐私保护与健康数据的二次利用：策略与方法。
Biomed Res Int. 2021 Oct 7;2021:6967166. doi: 10.1155/2021/6967166. eCollection 2021.
8
Real-World Data for Regulatory Decision Making: Challenges and Possible Solutions for Europe.用于监管决策的真实世界数据：欧洲面临的挑战与可能的解决方案
Clin Pharmacol Ther. 2019 Jul;106(1):36-39. doi: 10.1002/cpt.1426. Epub 2019 Apr 10.
9
Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen.欧美用于满足罕见病患者需求的孤儿药：预计将加大共同努力。
Orphanet J Rare Dis. 2017 Apr 3;12(1):64. doi: 10.1186/s13023-017-0617-1.
10
New pathway for qualification of novel methodologies in the European Medicines Agency.新的途径来确定欧洲药品管理局的新方法的资格。
Proteomics Clin Appl. 2011 Jun;5(5-6):248-55. doi: 10.1002/prca.201000130. Epub 2011 Apr 28.