新版《药物临床试验质量管理规范》背景下试验用药品管理的思考

Reflections on the management of investigational products in the light of the revised Good Clinical Practice

摘要

目的: 探讨在新版《药物临床试验质量管理规范》及ICH E6(R3)背景下试验用药品直达试验参与者这一新型试验用药品供应模式的应用现状、实施考量与落实措施,为优化其管理质量提供参考。方法: 通过文献分析,比较国内外试验用药品直达试验参与者模式应用的监管政策差异、实施现状及常见模式,分析实施的主要考量点。结果: 试验用药品直达试验参与者模式在国内外已有所应用,但缺乏统一的实施细则,在适当情况下可考虑使用,通过强化职责划分、伦理审查、知情同意、风险评估、资质审核、培训、信息化建设、风险管理、隐私保护及研究者监管等方面保障有效实施。结论: 试验用药品直达试验参与者模式符合“以患者为中心”理念,但尚处于探索阶段。计划使用时应在方案中充分论证其可行性,加强伦理审查、培训与风险管控,明确各方职责,从而保障参与者权益与数据可靠性。

Abstract

Objective: To explore the current application status, implementation considerations, and practical measures for a new model of delivering investigational drugs directly to trial participants (direct-to-subject, DTS) in the context of the revised Good Clinical Practice (GCP) and ICH E6 (R3), with the aim of providing a reference for optimizing its management quality. Methods: Through a literature review, this study compared regulatory policy differences, current implementation status, and common models for the direct-to-subject model between domestic and international settings, and analyze the key considerations for its implementation. Results: The direct-to-subject model has been adopted both domestically and internationally, but there is a lack of unified implementation guidelines. Its use may be considered when appropriate. Effective implementation can be ensured by strengthening the following aspects: clarification of responsibilities, ethical review, informed consent, risk assessment, qualification verification, training, information system development, risk management, privacy protection, and investigator oversight. Conclusion: The model aligns with the “patient-centered” philosophy but is still in its exploratory phase. When planning to adopt this approach, its feasibility should be fully demonstrated in the protocol, with enhanced emphasis on ethical review, training, and risk control, while clearly defining the responsibilities of all parties to safeguard participants' rights and ensure data reliability.

关键词

试验用药品管理 / 试验用药品直达试验参与者

Key words

investigational product management / direct-to-patient

[1] 张潇怡. 药物临床试验用药品全流程管理效果分析[J]. 交通医学, 2024, 38(6): 655-657.
[2] 张强, 席红领, 单爱莲. 临床试验用药品的管理探讨[J]. 中国临床药理学杂志, 2025, 41(2): 260-263.
[3] 续畅, 周心娜, 漆璐, 等. 基于文献计量学对我国临床试验用药品管理研究现状的数据挖掘[J]. 药学实践与服务, 2025, 43(8): 404-409.
[4] 王雅娟. JCI理念指导的信息化管理在临床试验用药品质量管理中的应用[J]. 临床研究, 2025, 33(8): 185-188.
[5] 周燕, 吕金鹏, 岳磊, 等. 某院临床试验用药品质量管理体系的构建[J]. 中国医药科学, 2023, 13(20): 167-171.
[6] 黎静波, 彭评志. 某机构临床试验用药品管理的现存问题及改进措施[J]. 中国处方药, 2024, 22(5): 53-56.
[7] 张晓方, 佟天琦, 徐岩, 等. 我国开展去中心化临床试验面临的主要风险和挑战及应对思考[J]. 中国食品药品监管, 2025(3): 70-81.
[8] 刘懿贤, 游佳, 邸相杰, 等. 去中心化临床试验(DCT)的创新与探索: 华西实践[J]. 中国医药导刊, 2024, 26(8): 774-779.
[9] 胡敏, 左泽锦, 吴维娟, 等. 药物临床试验直达患者(DTP)的药物运输模式探讨[J]. 中国食品药品监管, 2020(11): 94-101.
[10] 冷烨, 黄慧瑶, 于安琪, 等.中美欧临床试验药物直达患者监管策略比较及启示[J]. 癌症, 2023, 42(10): 542-551.
[11] 江柯萱. 新形势下我国去中心化临床试验的实践建议[J]. 现代医院, 2025, 25(4): 524-528.
[12] 季昭臣, 张俊华. “研究型药店”概念的提出及实践程序[J]. 天津中医药, 2025, 42(7): 860-864.
[13] DE JONG AJ, SANTA-ANA-TELLEZ Y, ZUIDGEEST MGP, et al. Direct-to-participant investigational medicinal product supply in clinical trials in Europe: Exploring the experiences of sponsors, site staff and couriers[J]. Brit J Clinical Pharma, 2023, 89(12): 3512-3522.
[14] HEATH M, DE JONG AJ, MAGORRIAN-SPENCE S, et al. The supply of investigational medicinal product and management of study materials for decentralized participants: insights from the Trials@HomeRADIALProof-of-concept trial[J]. Clin Pharma And Therapeutics, 2025, 118(5): 1079-1089.
[15] 肖爽, 李高扬, 董瑞华, 等. 去中心化临床试验设计和实施要点探析[J]. 中国新药杂志, 2024, 33(17): 1773-1777.
[16] 江旻, 刘晓红, 房虹, 等. 药物直达临床试验参与者模式在临床试验中实施的专家共识[J]. 中国新药杂志, 2025, 34(21): 2306-2315.
[17] 左旭, 黄樱硕, 李悦, 等. 去中心化药物临床试验伦理审查的实践探索[J]. 中国医学伦理学, 2025, 38(1): 40-45.
[18] 张晓方, 于冰, 龚瑛, 等. 中美欧药品监管机构去中心化临床试验指导原则比较研究[J]. 中国食品药品监管, 2024(8): 90-99.
[19] ROGERS A, DE PAOLI G, SUBBARAYAN S, et al. A systematic review of methods used to conduct decentralised clinical trials[J]. Brit J Clinical Pharma, 2022, 88(6): 2843-2862.
[20] 巫小文, 李昊翰, 陈秋琳, 等. 临床试验机构实施去中心化临床试验的挑战与策略研究[J]. 中国临床药学杂志, 2023, 32(12): 926-930.
[21] 崔欢欢, 唐凌, 崔灿, 等. 在罕见病药物临床研发中应用去中心化临床试验的考虑[J]. 罕见病研究, 2024, 3(2): 175-180.
[22] 卜丽娟, 江柯萱, 周吉银. 去中心化临床试验的挑战、对策及伦理审查要点[J]. 中国医学伦理学, 2024, 37(4): 399-407.

基金资助

2022年陆军军医大学人文社会科学基金重点项目(2022XRW02)