Sena Elena, Tacke Frank, Anstee Quentin M, Di Prospero Nicholas, Skalshøi-Kjær Mette, Muñoz-Martínez Sergio, Rivera-Esteban Jesús, Jiménez-Masip Alba, Bañales Jesús M, Martínez-Gómez María, Koenig Franz, Genescà Joan, Ratziu Vlad, Pericàs Juan M
Liver Unit, Vall d'Hebron University Hospital, Vall d'Hebron Institute for Research (VHIR), Universitat Autònoma de Barcelona, Spanish Network of Biomedical Research on Digestive and Liver Diseases (CIBERehd), Barcelona, Spain.
Department of Hepatology and Gastroenterology, Charité - Universitätsmedizin Berlin, Berlin, Germany.
Commun Med (Lond). 2024 Jul 16;4(1):144. doi: 10.1038/s43856-024-00560-5.
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.
欧盟以患者为中心的临床试验平台（EU-PEARL）项目（IMI2-853966）旨在开发工具，以建立综合研究平台（IRP），用于开展针对包括代谢功能障碍相关脂肪性肝炎（MASH）在内的各种疾病的适应性设计试验。成功的MASH IRP的一个关键组成部分是强大且可靠的临床研究网络（CRN）。在此，我们概述建立这样一个CRN所需的要素和预期步骤。
我们确定了可能作为MASH IRP和CRN基础的欧洲临床研究站点。向这些站点发送了一份调查问卷，以评估它们加入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。
总共邀请了141名研究人员参与调查，40%做出了回应。超过一半的回答（52%）将伴有晚期纤维化（F3-4）的MASH确定为需求未得到满足程度最高的亚组。关于确定试验候选人的困难程度，65%的人认为难度适中，30%的人认为非常困难。大多数受访者（94%）认为平台试验可以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%），22%的人表示他们的兴趣将取决于最初的行业资金。
虽然是初步结果，但我们关于做出回应的站点的调查结果对于为MASH IRP建立CRN的可能性而言是令人鼓舞的。然而，在短期情况下，提供MASH平台验证的资金计划和可持续性策略似乎是关键。
