Innovative methodologies for rare diseases clinical trials.
作者信息
Nabbout Rima, Hilgers Ralf-Dieter
机构信息
Reference centre for rare epilepsies Université Paris cité, Paris, France.
Assistance Publique-Hôpitaux de Paris, Hôpital Necker-Enfants Malades, Paris, France.
出版信息
Orphanet J Rare Dis. 2024 May 7;19(1):190. doi: 10.1186/s13023-024-03189-8.
Abstract
摘要
相似文献
1
Innovative methodologies for rare diseases clinical trials.罕见病临床试验的创新方法。
Orphanet J Rare Dis. 2024 May 7;19(1):190. doi: 10.1186/s13023-024-03189-8.
2
Composite endpoints, including patient reported outcomes, in rare diseases.包含患者报告结局在内的复合终点指标在罕见病中的应用。
Orphanet J Rare Dis. 2023 Sep 1;18(1):262. doi: 10.1186/s13023-023-02819-x.
3
Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspective.去中心化临床试验与罕见病：药品信息协会创新设计科学工作组（DIA-IDSWG）视角。
Orphanet J Rare Dis. 2023 Apr 11;18(1):79. doi: 10.1186/s13023-023-02693-7.
4
Innovative design and analysis for rare disease drug development.创新设计与罕见病药物开发分析。
J Biopharm Stat. 2020 May 3;30(3):537-549. doi: 10.1080/10543406.2020.1726371. Epub 2020 Feb 17.
5
Need to improve clinical trials in rare neurodegenerative disorders.需要改进罕见神经退行性疾病的临床试验。
Ann Ist Super Sanita. 2011;47(1):55-9. doi: 10.4415/ANN_11_01_12.
6
Characteristics and Public Availability of Results of Clinical Trials on Rare Diseases Registered at Clinicaltrials.gov.Clinicaltrials.gov 上注册的罕见病临床试验结果的特征及公开情况
JAMA Intern Med. 2016 Apr;176(4):556-8. doi: 10.1001/jamainternmed.2016.0137.
7
Methodology of clinical trials for rare diseases.罕见病临床试验方法学。
Best Pract Res Clin Rheumatol. 2014 Apr;28(2):247-62. doi: 10.1016/j.berh.2014.03.004.
8
Rare Gynecologic Tumors: Coming of Age.罕见妇科肿瘤：走向成熟。
Gynecol Oncol. 2020 Apr;157(1):1-2. doi: 10.1016/j.ygyno.2020.03.001.
9
Istore: a project on innovative statistical methodologies to improve rare diseases clinical trials in limited populations.Istore：一个关于创新统计方法的项目，旨在改善有限人群中的罕见病临床试验。
Orphanet J Rare Dis. 2024 Mar 2;19(1):96. doi: 10.1186/s13023-024-03103-2.
10
Editorial on special topic ISCB 2015: "Clinical research design, rare diseases, and personalized medicine".关于2015年国际计算生物学学会特别主题的社论：“临床研究设计、罕见病与个性化医疗”
Biom J. 2017 Jul;59(4):607-608. doi: 10.1002/bimj.201700065.
引用本文的文献
1
Results of health technology assessments of orphan drugs in Germany-lack of added benefit, evidence gaps, and persisting unmet medical needs.德国罕见病药物的卫生技术评估结果——缺乏额外益处、证据空白以及持续存在的未满足医疗需求
Int J Technol Assess Health Care. 2024 Dec 3;40(1):e68. doi: 10.1017/S026646232400062X.
本文引用的文献
1
Istore: a project on innovative statistical methodologies to improve rare diseases clinical trials in limited populations.Istore：一个关于创新统计方法的项目，旨在改善有限人群中的罕见病临床试验。
Orphanet J Rare Dis. 2024 Mar 2;19(1):96. doi: 10.1186/s13023-024-03103-2.
2
Applicability and added value of novel methods to improve drug development in rare diseases.新型方法在改善罕见病药物研发中的适用性和附加价值。
Orphanet J Rare Dis. 2018 Nov 12;13(1):200. doi: 10.1186/s13023-018-0925-0.
3
Recent advances in methodology for clinical trials in small populations: the InSPiRe project.小人群临床试验方法学的最新进展：InSPiRe 项目。
Orphanet J Rare Dis. 2018 Oct 25;13(1):186. doi: 10.1186/s13023-018-0919-y.
4
Lessons learned from IDeAl - 33 recommendations from the IDeAl-net about design and analysis of small population clinical trials.从 IDeAl 中吸取的教训——IDeal-net 关于小人群临床试验设计和分析的 33 条建议。
Orphanet J Rare Dis. 2018 May 11;13(1):77. doi: 10.1186/s13023-018-0820-8.
5
Development and content validation of a preliminary core set of patient- and caregiver-relevant outcomes for inclusion in a potential composite endpoint for Dravet Syndrome.用于纳入Dravet综合征潜在复合终点的患者及照料者相关初步核心结局集的开发与内容验证。
Epilepsy Behav. 2018 Jan;78:232-242. doi: 10.1016/j.yebeh.2017.08.029. Epub 2017 Nov 4.
6
Directions for new developments on statistical design and analysis of small population group trials.小群体试验统计设计与分析的新进展指南。
Orphanet J Rare Dis. 2016 Jun 14;11(1):78. doi: 10.1186/s13023-016-0464-5.
Zotero 插件