创新药物剂量探索优化中的临床药理学考量

Clinical Pharmacology Considerations in the Exploration and Optimization of Innovative Drug Dosing Regimens

摘要

创新药物的研发经历了从小分子药物到生物药物，再到细胞和基因治疗产品的重大变革。与此同时，对高质量医药产品的需求已不局限于药物创新类型，更加精准的治疗剂量也成为提高生命质量的重要一环。剂量探索不充分导致的药物临床试验失败案例也屡见不鲜，因此，创新药研发过程中的剂量探索优化至关重要。临床药理学研究贯穿于药物研发与上市后的全生命周期。通过深入探讨不同类型药物临床药理学特征及其在暴露-效应关系中关键指标选择、数据分析与证据评估等方面的科学考量，建立更加细致全面、多维度、以作用机制为中心的科学定量方法，阐述临床药理学研究在创新药物剂量探索优化以保障患者用药安全有效中的重要价值。

Abstract

The research and development of innovative drugs has undergone significant changes from small molecule drugs to biopharmaceuticals, and then to cell and gene therapy products. Meanwhile, the demand for high-quality pharmaceutical products is no longer limited to the types of innovative drugs; more precise dosing regimens have also become an important aspect in improving patients’ quality of life. In addition, the drug clinical trial failures due to insufficient dose exploration is also common, making dose exploration and optimization a crucial process in innovative drug development. Clinical pharmacology research spans the entire lifecycle from drug development to post-marketing surveillance. This article aims to establish a more refined, comprehensive, multidimensional, and mechanism-centered quantitative framework by delving into the clinical pharmacological characteristics of different types of drugs, along with scientific considerations in selecting critical metrics for exposure-response relationships, data analysis, and evidence evaluation, aiming to further elucidate the essential role of clinical pharmacological studies in exploring and optimizing innovative drug dosing to ensure safety and efficacy in patient care.

关键词

创新药 / 剂量探索优化 / 临床药理学

Key words

innovative drug / dose exploration and optimization / clinical pharmacology

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基金资助

药品监管科学全国重点实验室课题项目(2024SKLDRS0231)