设计综合早期协议以减少实质性修改,包括对患者队伍的考虑 - - 一个多利益相关方的共识观点,为德国的实践方法
R Schultz-Heienbrok1, S Baumann2, R Böhm3,4
1Charité Research Organisation GmbH, Berlin, Germany.
Frontiers in pharmacology
|February 11, 2026
概括
简化早期临床试验需要综合协议. 德国的框架使患者和健康志愿者的过渡能够无进行,提高了发展速度和透明度.
科学领域:
- 临床试验的设计
- 监管科学是一种监管科学.
- 药物开发 药物开发
背景情况:
- 实质性协议修改经常会延迟早期临床试验,并降低透明度.
- 在早期试验中整合健康的志愿者和患者会带来安全性,可行性和监管方面的挑战.
研究的目的:
- 确定在非高级治疗药物 (非ATMP),非瘤学研究的综合早期试验协议内无过渡的监管途径.
- 为协议设计,决策标准和治理提供可行的建议,以促进早期患者纳入.
主要方法:
- 德国进行了多方利益相关者共识进程.
- 对监管指导方针的分析,包括欧洲药物署 (EMA) 的第一人 (FIH) 指导方针.
- 开发协议过渡的"德国护"概念.
主要成果:
- 确定了监管途径,以便在集成协议中实现无过渡.
- 欧洲证券管理局FIH指南支持预先定义的过渡,具有明确的范围,安全门和决策规则.
- 德国护概念澄清了在哪些条件下过渡不被视为实质性修改,提高了规划确定性.
结论:
- 一个框架,包括德国护概念,可以在不影响安全性或合规性的情况下,早期将患者纳入非ATMP,非瘤学试验.
- 清晰的协议设计,决策标准和治理结构对于成功的综合试验至关重要.
- 在德国,报告了关于提出的框架的积极经验.
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