创新复方中药研发是中医药现代化和国际化的重要方向，其核心在于如何将传统中医药理论与现代药物研发技术有机结合，形成具有中国特色的新药研发体系。在这一过程中，量效关系研究是评价药物有效性和安全性的基础，也是确定临床用药剂量的关键环节。与化学药物相比，中药复方的量效关系研究面临着更为复杂的挑战。对临床试验与实际治疗中量效关系差异的成因进行分析；探讨创新复方中药研发中的量效关系设计、确证性临床试验中剂量选择的决定因素，进而提出临床前药效学研究方法、探索性临床试验设计、确证性临床试验设计以及上市后监测与再评价的优化策略，为提高临床试验与实际治疗中剂量选择的准确性提供参考，促进创新复方中药的研发和应用。

The development of innovative compound of traditional Chinese medicine（TCM） is a crucial direction for the modernization and internationalization of TCM. Its core lies in effectively integrating traditional TCM theory with modern drug research and development（R&D） technologies to establish a new drug development system with Chinese characteristics. In this process, research on the dose-effect relationship serves as the foundation for evaluating drug efficacy and safety and is a key step in determining clinical dosages. Compared to chemical drugs, dose-effect relationship studies for compound of TCM face more complex challenges. This paper analyzes the causes of discrepancies between the dose-effect relationships observed in clinical trials and those in actual clinical practice. It explores the design considerations for dose-effect relationships in innovative compound of TCM R&D and the determinants of dosage selection in confirmatory clinical trials. Furthermore, it proposes optimization strategies for preclinical pharmacodynamic study methods, exploratory clinical trial design, confirmatory clinical trial design, and post-marketing surveillance and re-evaluation. The aim is to provide references for improving the accuracy of dosage selection in clinical trials and actual treatment, thereby promoting the development and application of innovative Chinese herbal compound formulas.

R285.5

四大慢病重大专项(2023ZD0509300)