绘制细胞和基因治疗产品的临床发展轨迹
Magdi Elsallab1,2, Michelle Ouvina3, Andrea Arfe4
1Harvard-MIT Center for Regulatory Science, Harvard Medical School, Boston, Massachusetts, USA.
Clinical pharmacology and therapeutics
|December 10, 2024
概括
细胞和基因疗法 (CGTs) 是有希望的,但面临着发展的不确定性. 这项研究发现,CGT的整体批准概率很低,为5.3%,根据孤儿状态和适应症的变化.
科学领域:
- 生物技术是生物技术.
- 制药发展 制药发展
- 监管科学 监管科学
背景情况:
- 细胞和基因疗法 (CGTs) 为未满足的医疗需求提供了新的治疗途径.
- 对于制药公司来说,CGT复杂和非传统的发展途径带来了重大风险.
- 了解CGT的成功率对于战略投资和发展规划至关重要.
研究的目的:
- 评估CGT产品的临床开发成功率.
- 估计临床试验阶段过渡的概率和对CGT的监管批准.
- 确定影响CGT发展计划成功的因素.
主要方法:
- 分析了995个CGT产品和1961个发展计划,从1993年到2023年.
- 包括在美国,欧洲,日本,加拿大和瑞士销售的产品.
- 检查产品特征 (例如,孤儿名称,指示) 与批准可能性之间的关联,包括CAR T和AAV基因疗法的子分析.
主要成果:
- 对CGT的监管批准的总体概率为5.3%.
- 与非孤儿 (3.2%) 相比,被指定为孤儿的发展计划的批准概率更高 (9.4%).
- 瘤学指标的批准概率 (3.2%) 低于非瘤学指标 (8.0%). 对于CAR T和AAV基因疗法,批准概率为13.6%.
结论:
- 总体而言,CGT产品获得监管机构批准的概率很低.
- 孤儿身份和治疗区域显著影响CGT的成功率.
- 特定的CGT类型,如CAR T和AAV基因疗法,表现出不同的,尽管更高的批准概率.
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