Duke Clinical Research Institute, Duke University School of Medicine, Box 2834, Durham, NC, 27701, USA.
Microsoft Research, Cambridge, MA, USA.
Trials. 2021 Aug 16;22(1):537. doi: 10.1186/s13063-021-05489-x.
BACKGROUND: Interest in the application of machine learning (ML) to the design, conduct, and analysis of clinical trials has grown, but the evidence base for such applications has not been surveyed. This manuscript reviews the proceedings of a multi-stakeholder conference to discuss the current and future state of ML for clinical research. Key areas of clinical trial methodology in which ML holds particular promise and priority areas for further investigation are presented alongside a narrative review of evidence supporting the use of ML across the clinical trial spectrum. RESULTS: Conference attendees included stakeholders, such as biomedical and ML researchers, representatives from the US Food and Drug Administration (FDA), artificial intelligence technology and data analytics companies, non-profit organizations, patient advocacy groups, and pharmaceutical companies. ML contributions to clinical research were highlighted in the pre-trial phase, cohort selection and participant management, and data collection and analysis. A particular focus was paid to the operational and philosophical barriers to ML in clinical research. Peer-reviewed evidence was noted to be lacking in several areas. CONCLUSIONS: ML holds great promise for improving the efficiency and quality of clinical research, but substantial barriers remain, the surmounting of which will require addressing significant gaps in evidence.
背景:对机器学习 (ML) 在临床试验的设计、实施和分析中的应用的兴趣日益浓厚,但此类应用的证据基础尚未得到调查。本文回顾了一次多方利益攸关者会议的会议记录,以讨论机器学习在临床研究中的现状和未来。提出了 ML 在临床试验方法学的特定领域具有特别的前景,以及进一步研究的优先领域,并对支持在整个临床试验范围内使用 ML 的证据进行了叙述性综述。
结果:会议与会者包括利益攸关方,如生物医学和 ML 研究人员、美国食品和药物管理局 (FDA) 的代表、人工智能技术和数据分析公司、非营利组织、患者权益团体和制药公司。在临床试验前阶段、队列选择和参与者管理以及数据收集和分析中突出了 ML 对临床研究的贡献。特别关注了 ML 在临床研究中的操作和哲学障碍。注意到在几个领域缺乏同行评审证据。
结论:ML 为提高临床研究的效率和质量带来了巨大的希望,但仍存在重大障碍,要克服这些障碍,就需要解决证据方面的重大差距。
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