Office of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
CPT Pharmacometrics Syst Pharmacol. 2021 Dec;10(12):1479-1484. doi: 10.1002/psp4.12709. Epub 2021 Nov 3.
Quantitative systems pharmacology (QSP) has been proposed as a scientific domain that can enable efficient and informative drug development. During the past several years, there has been a notable increase in the number of regulatory submissions that contain QSP, including Investigational New Drug Applications (INDs), New Drug Applications (NDAs), and Biologics License Applications (BLAs) to the US Food and Drug Administration. However, there has been no comprehensive characterization of the nature of these regulatory submissions regarding model details and intended applications. To address this gap, a landscape analysis of all the QSP submissions as of December 2020 was conducted. This report summarizes the (1) yearly trend of submissions, (2) proportion of submissions between INDs and NDAs/BLAs, (3) percentage distribution along the stages of drug development, (4) percentage distribution across various therapeutic areas, and (5) nature of QSP applications. In brief, QSP is increasingly applied to model and simulate both drug effectiveness and safety throughout the drug development process across disease areas.
定量系统药理学（QSP）已被提议作为一个能够实现高效和信息丰富的药物开发的科学领域。在过去的几年中，向美国食品和药物管理局（FDA）提交的包含 QSP 的监管文件数量显著增加，包括新药临床试验申请（IND）、新药申请（NDA）和生物制品许可申请（BLA）。然而，对于这些监管文件中关于模型细节和预期应用的性质，还没有进行全面的描述。为了弥补这一空白，对截至 2020 年 12 月的所有 QSP 提交进行了景观分析。本报告总结了（1）提交的年度趋势，（2）IND 和 NDA/BLA 之间的提交比例，（3）药物开发各个阶段的分布百分比，（4）各个治疗领域的分布百分比，以及（5）QSP 应用的性质。简而言之，QSP 越来越多地应用于模拟和预测整个药物开发过程中药物的有效性和安全性，涉及各个疾病领域。
