Challenges and opportunities for Multi-National Investigator-Initiated clinical trials for ALS: European and United States collaborations.
Challenges and opportunities for Multi-National Investigator-Initiated clinical trials for ALS: European and United States collaborations.
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DOI：
10.1080/21678421.2021.1879866
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发表时间：
2021-08
影响因子：
2.8
通讯作者：
Benatar M
中科院分区：
文献类型：
--
作者：
Lingor P;Koch JC;Statland JM;Hussain S;Hennecke C;Wuu J;Langbein T;Ahmed R;Günther R;Ilse B;Kassubek J;Kollewe K;Kuttler J;Leha A;Lengenfeld T;Meyer T;Neuwirth C;Tostmann R;Benatar M
旨在测试肌萎缩侧索硬化症（ALS）患者实验性治疗方法疗效的临床试验的一个固有挑战是该疾病的相对罕见性。一个有前途的解决方案，这个问题是一个多中心的方法，理想情况下，包括分布在广泛的地理区域的网站。为了支持这种方法，欧洲E-RARE计划和美国国立卫生研究院（NIH）合作支持研究者发起的ROCK-ALS试验（Eudra-CT-Nr.：2017-003676-31，NCT 03792490）作为欧洲和北美中心之间的跨国合作，由欧洲研究者领导。然而，在这一国际审判的筹备过程中，出现了一些意想不到的法律的、行政和财务复杂问题，需要对拟议的审判计划进行重大调整。在这里，我们报告了我们克服这些障碍的经验，并描述了我们探索的潜在解决方案。我们的经验可能会为将来实施涉及欧洲和美国中心的跨国制药商发起的试验提供信息。
An inherent challenge to clinical trials that aim to test the efficacy of experimental therapeutics for patients with amyotrophic lateral sclerosis (ALS) is the relative rarity of the disease. A promising solution to this problem is a multi-center approach that ideally includes sites distributed across a broad geographic area. In support of such an approach, the European E-RARE program and the United States National Institutes of Health (NIH) partnered to support the investigator-initiated ROCK-ALS trial (Eudra-CT-Nr.: 2017-003676-31, NCT03792490) as a multi-national collaboration between centers in Europe and North America that is led by a European investigator. During the set-up of this international trial, however, a number of unanticipated legal, administrative, and financial complexities emerged that required significant adaptation of the proposed trial scheme. Here, we report our experience navigating these obstacles and describe the potential solutions that we explored. Our experience may inform future efforts to implement multi-national investigator-initiated trials that involve both European and United States centers.
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