Suppr超能文献

RD-LIVES:一种用于罕见病治疗疗效和安全性的循证合成系统。

RD-LIVES: A Living Evidence Synthesis System for Rare Disease Treatment Efficacy and Safety.

作者信息

Wang Jinlian, Li Hui, Liu Hongfang

机构信息

The McWilliams School of Biomedical Informatics, Houston, TX, USA.

出版信息

AMIA Jt Summits Transl Sci Proc. 2025 Jun 10;2025:607-613. eCollection 2025.

Abstract

Although rare diseases (RD) are gaining priority in healthcare worldwide, developing research policies for studying them in public settings remains challenging due to the limited evidence available. Evidence generation is crucial for rare diseases, requiring systematic assessment of study quality across multiple sources. Given the scarcity of patients, literature and clinical trial data for orphan drugs, we developed RD-LIVES-a tool designed to automatically accelerate evidence collection from literature and clinical trials for systematic reviews and meta-analyses. This tool enhances our understanding of treatment outcomes, determines appropriate follow-up durations, and informs the required treatment impact size for new drugs. Using Idiopathic Pulmonary Fibrosis (IPF) as an example, we demonstrate how RD-LIVES automates evidence collection and element extraction. The results indicate that RD-LIVES plays a vital role in designing costly prospective trials and has the potential to increase the likelihood of successful trial outcomes.

摘要

尽管罕见病在全球医疗保健领域正日益受到重视,但由于可用证据有限,制定在公共环境中研究罕见病的研究政策仍具有挑战性。证据生成对于罕见病至关重要,需要对多个来源的研究质量进行系统评估。鉴于孤儿药的患者、文献和临床试验数据稀缺,我们开发了RD-LIVES——一种旨在自动加速从文献和临床试验中收集证据以进行系统评价和荟萃分析的工具。该工具增强了我们对治疗结果的理解,确定了适当的随访持续时间,并为新药所需的治疗影响大小提供了信息。以特发性肺纤维化(IPF)为例,我们展示了RD-LIVES如何自动收集证据和提取要素。结果表明,RD-LIVES在设计成本高昂的前瞻性试验中发挥着至关重要的作用,并且有可能增加试验成功的可能性。

相似文献

1
RD-LIVES: A Living Evidence Synthesis System for Rare Disease Treatment Efficacy and Safety.
AMIA Jt Summits Transl Sci Proc. 2025 Jun 10;2025:607-613. eCollection 2025.
3
The future of Cochrane Neonatal.
Early Hum Dev. 2020 Nov;150:105191. doi: 10.1016/j.earlhumdev.2020.105191. Epub 2020 Sep 12.
8
Pirfenidone: a novel agent for the treatment of idiopathic pulmonary fibrosis.
Ann Pharmacother. 2013 Mar;47(3):361-7. doi: 10.1345/aph.1R337. Epub 2013 Feb 12.
9
Pirfenidone in idiopathic pulmonary fibrosis: real-life experience in the referral centre of Siena.
Ther Adv Respir Dis. 2020 Jan-Dec;14:1753466620906326. doi: 10.1177/1753466620906326.

本文引用的文献

1
Quantifying absolute benefit for adjuvant treatment options in renal cell carcinoma: A living interactive systematic review and network meta-analysis.
Crit Rev Oncol Hematol. 2022 Jul;175:103706. doi: 10.1016/j.critrevonc.2022.103706. Epub 2022 May 7.
2
Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.
Nat Rev Drug Discov. 2020 Feb;19(2):93-111. doi: 10.1038/s41573-019-0049-9. Epub 2019 Dec 13.
3
Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database.
Eur J Hum Genet. 2020 Feb;28(2):165-173. doi: 10.1038/s41431-019-0508-0. Epub 2019 Sep 16.
5
Challenges of developing and conducting clinical trials in rare disorders.
Am J Med Genet A. 2018 Apr;176(4):773-783. doi: 10.1002/ajmg.a.38413. Epub 2017 Aug 16.
6
History of Orphan Drug Regulation-United States and Beyond.
Clin Pharmacol Ther. 2016 Oct;100(4):342-3. doi: 10.1002/cpt.426. Epub 2016 Aug 26.
8
A phase 3 trial of pirfenidone in patients with idiopathic pulmonary fibrosis.
N Engl J Med. 2014 May 29;370(22):2083-92. doi: 10.1056/NEJMoa1402582. Epub 2014 May 18.
9
Systematic review of available evidence on 11 high-priced inpatient orphan drugs.
Orphanet J Rare Dis. 2013 Aug 16;8:124. doi: 10.1186/1750-1172-8-124.
10
What is wrong with orphan drug policies?
Value Health. 2012 Dec;15(8):1185-91. doi: 10.1016/j.jval.2012.09.004.

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验