Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
Center for Translational Medicine, University of Maryland School of Pharmacy, Baltimore, Maryland, USA.
Clin Pharmacol Ther. 2024 Apr;115(4):673-686. doi: 10.1002/cpt.3152. Epub 2024 Jan 3.
Technological innovations, such as artificial intelligence (AI) and machine learning (ML), have the potential to expedite the goal of precision medicine, especially when combined with increased capacity for voluminous data from multiple sources and expanded therapeutic modalities; however, they also present several challenges. In this communication, we first discuss the goals of precision medicine, and contextualize the use of AI in precision medicine by showcasing innovative applications (e.g., prediction of tumor growth and overall survival, biomarker identification using biomedical images, and identification of patient population for clinical practice) which were presented during the February 2023 virtual public workshop entitled "Application of Artificial Intelligence and Machine Learning for Precision Medicine," hosted by the US Food and Drug Administration (FDA) and University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI). Next, we put forward challenges brought about by the multidisciplinary nature of AI, particularly highlighting the need for AI to be trustworthy. To address such challenges, we subsequently note practical approaches, viz., differential privacy, synthetic data generation, and federated learning. The proposed strategies - some of which are highlighted presentations from the workshop - are for the protection of personal information and intellectual property. In addition, methods such as the risk-based management approach and the need for an agile regulatory ecosystem are discussed. Finally, we lay out a call for action that includes sharing of data and algorithms, development of regulatory guidance documents, and pooling of expertise from a broad-spectrum of stakeholders to enhance the application of AI in precision medicine.
技术创新,如人工智能 (AI) 和机器学习 (ML),具有加速精准医学目标的潜力,尤其是与从多个来源增加大容量数据的能力以及扩大治疗方式相结合时;然而,它们也带来了一些挑战。在本通讯中,我们首先讨论精准医学的目标,并通过展示在 2023 年 2 月题为“人工智能和机器学习在精准医学中的应用”的虚拟公开研讨会上展示的创新应用(例如,肿瘤生长和总生存预测、使用生物医学图像识别生物标志物,以及为临床实践确定患者人群)来阐述 AI 在精准医学中的应用,该研讨会由美国食品和药物管理局 (FDA) 和马里兰卓越监管科学与创新中心 (M-CERSI) 主办。接下来,我们提出了 AI 多学科性质带来的挑战,特别是强调了 AI 需要值得信赖。为了解决这些挑战,我们随后指出了一些实用的方法,即差分隐私、合成数据生成和联邦学习。所提出的策略——其中一些是研讨会的重点介绍——是为了保护个人信息和知识产权。此外,还讨论了基于风险的管理方法以及需要灵活的监管生态系统等方法。最后,我们呼吁采取行动,包括共享数据和算法、制定监管指导文件以及汇集来自广泛利益相关者的专业知识,以增强 AI 在精准医学中的应用。