Needs assessment for creation of a platform trial network in metabolic-dysfunction associated steatohepatitis

IF 5.4 Q1 MEDICINE, RESEARCH & EXPERIMENTAL
Elena Sena, Frank Tacke, Quentin M. Anstee, Nicholas Di Prospero, Mette Skalshøi- Kjær, Sergio Muñoz-Martínez, Jesús Rivera-Esteban, Alba Jiménez-Masip, Jesús M. Bañales, María Martínez-Gómez, Franz Koenig, Joan Genescà, Vlad Ratziu, Juan M. Pericàs
{"title":"Needs assessment for creation of a platform trial network in metabolic-dysfunction associated steatohepatitis","authors":"Elena Sena, Frank Tacke, Quentin M. Anstee, Nicholas Di Prospero, Mette Skalshøi- Kjær, Sergio Muñoz-Martínez, Jesús Rivera-Esteban, Alba Jiménez-Masip, Jesús M. Bañales, María Martínez-Gómez, Franz Koenig, Joan Genescà, Vlad Ratziu, Juan M. Pericàs","doi":"10.1038/s43856-024-00560-5","DOIUrl":null,"url":null,"abstract":"The EU Patient-cEntric clinicAl tRial pLatforms (EU-PEARL) project (IMI2-853966) aimed to develop tools to establish integrated research platforms (IRP) for conducting adaptive-design trials in various diseases, including metabolic-dysfunction associated steatohepatitis (MASH). One essential component of a successful MASH IRP is a robust and reliable Clinical Research Network (CRN). Herein, we outline the required elements and anticipated steps to set-up such a CRN. We identified European clinical research sites that could potentially serve as the foundation for MASH IRP and a CRN. A survey was sent to sites to assess their interest in joining a CRN, their familiarity with platform trials, and their capacity to participate in a future MASH IRP. A total of 141 investigators were invited to participate in the survey, and 40% responded. More than half of the answers (52%) identify MASH with advanced fibrosis (F3-4) as the subpopulation with the greatest unmet need. Regarding the difficulty in identifying candidates for trials, 65% find it is moderately difficult and 30% very difficult. Most respondents (94%) believe that a platform trial could offer substantial benefits to patients. Nearly all researchers express interest in participating in a platform trial (78%), with 22% indicating their interest would be contingent on initial industry funding. While preliminary, our findings on responding sites are encouraging for the potential establishment of a CRN for a MASH IRP. However, funding schemes and sustainability strategies to provide proof-of-platform in MASH seem key in the short-term scenario. Metabolic dysfunction-associated steatohepatitis (MASH) occurs when the liver becomes damaged due to the build up of fat, which is often related to obesity and diabetes. There is a lack of effective drug treatments for MASH, so strategies to strengthen clinical research in this area are needed. Here, we survey key European experts on MASH to assess their interest in joining a network of MASH researchers and their interest in participating in a new type of clinical trial called a platform trial, where multiple drugs can be tested simultaneously. Researchers largely agree that these are promising approaches to boost drug development in the field, although have concerns regarding funding and sustainability strategies. Our findings may inform the creation of a network of MASH researchers capable of running a platform trial, which in turn may speed up research into treatments for MASH. Sena et al. survey European clinical research sites that could potentially serve as the foundation for a MASH integrated research platform and clinical research network. Respondents generally believe that a MASH platform trial could benefit patients and are interested in participating, though securing industry funding is identified as a priority.","PeriodicalId":72646,"journal":{"name":"Communications medicine","volume":null,"pages":null},"PeriodicalIF":5.4000,"publicationDate":"2024-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s43856-024-00560-5.pdf","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Communications medicine","FirstCategoryId":"1085","ListUrlMain":"https://www.nature.com/articles/s43856-024-00560-5","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}
引用次数: 0

Abstract

The EU Patient-cEntric clinicAl tRial pLatforms (EU-PEARL) project (IMI2-853966) aimed to develop tools to establish integrated research platforms (IRP) for conducting adaptive-design trials in various diseases, including metabolic-dysfunction associated steatohepatitis (MASH). One essential component of a successful MASH IRP is a robust and reliable Clinical Research Network (CRN). Herein, we outline the required elements and anticipated steps to set-up such a CRN. We identified European clinical research sites that could potentially serve as the foundation for MASH IRP and a CRN. A survey was sent to sites to assess their interest in joining a CRN, their familiarity with platform trials, and their capacity to participate in a future MASH IRP. A total of 141 investigators were invited to participate in the survey, and 40% responded. More than half of the answers (52%) identify MASH with advanced fibrosis (F3-4) as the subpopulation with the greatest unmet need. Regarding the difficulty in identifying candidates for trials, 65% find it is moderately difficult and 30% very difficult. Most respondents (94%) believe that a platform trial could offer substantial benefits to patients. Nearly all researchers express interest in participating in a platform trial (78%), with 22% indicating their interest would be contingent on initial industry funding. While preliminary, our findings on responding sites are encouraging for the potential establishment of a CRN for a MASH IRP. However, funding schemes and sustainability strategies to provide proof-of-platform in MASH seem key in the short-term scenario. Metabolic dysfunction-associated steatohepatitis (MASH) occurs when the liver becomes damaged due to the build up of fat, which is often related to obesity and diabetes. There is a lack of effective drug treatments for MASH, so strategies to strengthen clinical research in this area are needed. Here, we survey key European experts on MASH to assess their interest in joining a network of MASH researchers and their interest in participating in a new type of clinical trial called a platform trial, where multiple drugs can be tested simultaneously. Researchers largely agree that these are promising approaches to boost drug development in the field, although have concerns regarding funding and sustainability strategies. Our findings may inform the creation of a network of MASH researchers capable of running a platform trial, which in turn may speed up research into treatments for MASH. Sena et al. survey European clinical research sites that could potentially serve as the foundation for a MASH integrated research platform and clinical research network. Respondents generally believe that a MASH platform trial could benefit patients and are interested in participating, though securing industry funding is identified as a priority.

Abstract Image

建立代谢功能障碍相关性脂肪性肝炎平台试验网络的需求评估。
项目背景:欧盟患者-临床-综合研究平台(EU-PEARL)项目(IMI2-853966)旨在开发建立综合研究平台(IRP)的工具,以开展包括代谢功能障碍相关性脂肪性肝炎(MASH)在内的各种疾病的适应性设计试验。成功的 MASH IRP 的一个重要组成部分是强大可靠的临床研究网络(CRN)。在此,我们概述了建立这样一个 CRN 所需的要素和预期步骤:我们确定了有可能作为 MASH IRP 和 CRN 基础的欧洲临床研究机构。我们向这些研究机构发送了一份调查问卷,以评估它们对加入 CRN 的兴趣、对平台试验的熟悉程度以及参与未来 MASH IRP 的能力:共有 141 名调查员受邀参与调查,40% 的调查员做出了回应。半数以上(52%)的回答者认为,患有晚期纤维化(F3-4)的 MASH 是需求未得到最大满足的亚群。关于确定试验候选者的难度,65% 的人认为难度适中,30% 的人认为难度很大。大多数受访者(94%)认为,平台试验可为患者带来巨大益处。几乎所有研究人员都表示有兴趣参与平台试验(78%),其中 22% 表示他们的兴趣将取决于最初的行业资助:虽然是初步的,但我们对响应机构的研究结果令人鼓舞,有可能为 MASH IRP 建立一个 CRN。然而,在短期内,为 MASH 提供平台验证的资助计划和可持续发展战略似乎是关键所在。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
自引率
0.00%
发文量
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信