非酒精性脂肪性肝炎新药临床试验:设计和实践中的挑战

茅益民

doi:10.3969/j.issn.1001-5256.2017.12.007

非酒精性脂肪性肝炎新药临床试验:设计和实践中的挑战

DOI: 10.3969/j.issn.1001-5256.2017.12.007

茅益民

上海交通大学医学院附属仁济医院,上海市消化疾病研究所,上海市脂肪性肝病诊治研究中心
上海交通大学医学院临床研究中心

基金项目:

十三五科技重大专项(2017ZX09304016)；国家自然科学基金(81670524)；上海交通大学医学院多中心临床研究项目资助；上海申康医院发展中心临床创新三年行动计划资助；

详细信息

中图分类号: R575.5
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出版历程
- 出版日期: 2017-12-20

Clinical trials of investigational new drugs for nonalcoholic steatohepatitis:challenges in design and practice

Mao YiMin

Research funding:

摘要

摘要: 针对非酒精性脂肪性肝病的新药研发目前是肝病领域的研究热点之一。然而,在开展Ⅱ/Ⅲ期临床试验时,无论是方案设计层面还是实施层面,都面临很大的挑战。由于学术界对非酒精性脂肪性肝炎组织学的确切定义尚存有争议,而且目前尚缺乏大规模长期基于非酒精性脂肪性肝病/非酒精性脂肪性肝炎临床病理学的自然史研究,因此,临床试验中研究人群的选择和界定、研究终点的选择及其真正临床价值的解释等都面临很大的挑战。而且这类临床试验中的混杂因素复杂,需在方案设计和实施层面尽可能控制,以最大程度降低对研究结论可能带来的偏倚。
- 非酒精性脂肪性肝炎 /
- 临床试验 /
- 药物设计
Abstract: Research and development of investigational new drugs for nonalcoholic fatty liver disease is now a research hotspot in the field of liver disease. However, there are still great challenges in protocol design and implementation in phase II/III clinical trials. Since there are still controversies over the exact histological definition of nonalcoholic steatohepatitis in the academic world and a lack of long-term large-scale studies on the natural history and clinical pathology of nonalcoholic fatty liver disease/nonalcoholic steatohepatitis, we still face a lot of challenges in the selection and definition of study population, selection of study endpoints, and interpretation of true clinical value. In addition, such clinical trials often have various confounding factors, which should be controlled as much as possible in protocol design and implementation, in order to minimize the bias in research conclusions.
- nonalcoholic steatohepatitis /
- clinical trial /
- drug design

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参考文献(9)

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