在儿科的药物治疗中应对监管挑战:通往安全有效治疗的途径
Gianmichele Villano1, Gianluca D'Onofrio1, Antonella Riva1,2
1Department of Neurosciences Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Genoa, Italy.
由于高成本和监管障碍,儿童药物开发面临着挑战. 以科学为导向的方法优化激励措施对于开发新的抗发作药物 (ASM) 对于儿童至关重要.
科学领域:
- 神经学 神经学
- 药理学 药理学是指药理学的学科.
- 监管科学 监管科学
背景情况:
- 是一种常见的儿童神经疾病,但在药物开发中,儿童往往是"治疗孤儿".
- 为儿童群体开发新的抗发作药物 (ASM) 提出了重大的财务和监管挑战.
- 由于感知到的风险和融资问题,对开发新的ASM的商业兴趣正在下降.
研究的目的:
- 审查小儿症药物开发的历史背景.
- 分析美国食品和药物管理局 (FDA) 和欧洲药品管理局 (EMA) 对ASM的现有监管框架和激励措施.
- 确定加速开发儿童创新性治疗方法的策略.
主要方法:
- 采用了叙事审查方法.
- 在PubMed,EMBASE,Scopus和谷歌学者中使用相关关键字进行文献搜索.
- 官方的FDA和EMA网站和文件也被系统地选.
主要成果:
- 该评论追踪了儿科药物开发在的演变.
- 分析了目前的FDA和EMA监管框架以及影响儿童ASM批准的激励措施.
- 该研究强调了优化激励措施以满足儿科需求的关键需求.
结论:
- 更专注的,以科学为导向的方法,尽量减少非科学影响,可以加速治疗创新.
- 调整研究激励与特定的儿科综合征和适合年龄的药理学是必不可少的.
- 解决儿科药物开发中的挑战对于改善儿童的结果至关重要.
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