高风险神经母细胞瘤治疗的向疗法:需要整合监管和科学工具
Adriana Ceci1, Rosa Conte1, Antonella Didio1
1Research Department, Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus, Bari, Italy.
Frontiers in medicine
|July 31, 2023
概括
开发新的神经母细胞瘤治疗方法面临监管障碍. 对活性物质的审查发现了许多早期试验,但很少获得监管批准,凸显了改善儿科药物开发模型的需要.
科学领域:
- 在瘤学瘤学.
- 儿科瘤学 儿科瘤学
- 药物开发 药物开发
背景情况:
- 神经母细胞瘤 (NBL) 治疗需要新型活性物质 (AS).
- 现有的监管途径为儿科药物开发带来了挑战.
研究的目的:
- 评估针对NBL的附属系统的发展和监管状态.
- 确定监管差距,并建议改善儿科NBL产品开发.
主要方法:
- 对临床前研究,临床试验 (CT),营销许可和监管程序 (例如,PIP,豁免,孤儿指定) 的系统审查.
- 对188个已识别的NBL附属系统进行分析.
主要成果:
- 70个AS有283个CT,其中52%处于早期阶段 (PK/PD/剂量).
- 有限的第三阶段研究;只有14个AS完成了研究.
- 37个AS获得了儿科调查计划 (PIP),14个专门用于NBL;只有两个获得了儿科指示扩展.
结论:
- 很少有AS通过监管过程获得儿科指示.
- 需要一个更有效的儿科发育模型,整合临床前和创新的临床设计.
- 加强科学和监管努力的整合对于儿科NBL药物开发至关重要.
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