在药物开发协议中利用多基因风险评分
Michelle A Pressly1, Robert Schuck1, Padmaja Mummaneni1
1Division of Translational and Precision Medicine, Office of Clinical Pharmacology, Office of Translational Science, Center for Drug Evaluation and Research, U.S. Food & Drug Administration, Silver Spring, MD, USA.
Pharmacogenomics
|April 9, 2025
概括
多基因风险评分 (PRS) 越来越多地指导药物开发中的临床试验,特别是在神经学和瘤学领域. 它们的使用,虽然不太常见,但支持早期试验,需要进一步研究.
科学领域:
- 药物基因组学 药物基因组学
- 临床试验设计 临床试验设计
- 生物统计学 生物统计学
背景情况:
- 多基因风险评分 (PRSs) 利用遗传变异来评估疾病风险.
- PRS在药物开发中具有潜在的应用,包括临床试验丰富和治疗反应预测.
研究的目的:
- 分析在提交给食品和药物管理局 (FDA) 的临床试验中PRS的不断发展的使用情况.
- 确定PRS最常被应用的治疗领域和试验阶段.
主要方法:
- 对FDA提交文件的分析,以随着时间的推移跟踪PRS的使用情况.
- 根据治疗领域和开发阶段对临床试验方案的分类.
主要成果:
- 观察到PRS用于指导临床试验的应用趋势越来越大.
- PRS主要用于神经病学,放射学,精神病学和瘤学.
- 大多数应用发生在早期阶段试验 (第1,1/2,3阶段) 中,用于二次或探索性分析.
- 大约一半的协议涉及新型PRS开发,而另一半则利用现有PRS.
结论:
- 在临床试验中PRS的日益应用凸显了它们在药物开发中的新兴作用.
- 需要进一步的研究,以充分了解其影响,并优化PRS在临床研究中的使用.
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