一个路线图,以以人为中心对先进治疗药物的安全性评估
Hsiao-Tzu Chien1, Victoria C de Leeuw2, Joantine C J van Esterik2
1Medicines Evaluation Board, Utrecht, the Netherlands; Radboud University Medical Center, Nijmegen, the Netherlands.
Regulatory toxicology and pharmacology : RTP
|April 22, 2024
概括
利益相关者采访显示,虽然体内研究是先进治疗药物 (ATMP) 的标准,但存在挑战. 通过使用新方法方法 (NAMs) 制定了以人为中心的安全评估路线图.
科学领域:
- 制药科学 制药科学
- 毒理学 毒理学 毒理学
- 生物技术是生物技术.
背景情况:
- 先进治疗药物 (ATMP) 在非临床开发中存在独特的挑战,原因是高的人类特异性和物种间差异影响了体内研究的相关性.
- 目前对ATMPs的体内研究的监管依赖面临局限性,需要探索替代方法.
- 了解利益相关者的观点对于推进新疗法非临床安全性评估策略至关重要.
研究的目的:
- 探索利益相关者的观点,在ATMP开发中使用体内研究.
- 确定ATMPs非临床开发的障碍和潜在解决方案.
- 建立以人为中心的ATMP安全评估的路线图.
主要方法:
- 与参与ATMP开发的9个国家的17个利益相关者进行了半结构化访谈.
- 与关键利益相关者组织了一场研讨会,讨论确定的主题.
- 定性数据分析为制定战略路线图提供了信息.
主要成果:
- 进行体内研究仍然是ATMP开发的既定做法.
- 主要障碍包括定义临床前数据要求和优化有限的人类样本的使用.
- 有一个公认的需要改进新方法方法论 (NAMs) 的应用和整合与体内研究.
结论:
- 制定了一份以人为中心的ATMP安全评估路线图,强调数据透明度和NAM的进步.
- 未来的ATMP开发需要一个平衡的方法,将传统的体内研究与验证的NAM集成在一起.
- 为了在监管决策中有效实施NAM,对NAM的持续评估和合格至关重要.
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