超越数字:用于监管和卫生技术评估 (HTA) 决策的试验访谈数据的用途和价值
Carla Dias-Barbosa1, Miriam Kimel2, Paulina Rolska-Wojcik3
1PPD™ Evidera™ Patient-Centered Research, Thermo Fisher Scientific, London, UK. carla.dias-barbosa@thermofisher.com.
定性试验研究 (ITR) 在监管和医疗技术评估 (HTA) 决策方面越来越重要. 然而,其在FDA和EMA批准药物的使用仍然有限,需要早期规划以获得更大的影响.
科学领域:
- 医疗保健服务研究 医疗服务研究
- 监管科学 监管科学
- 患者报告的结果
背景情况:
- 美国食品和药物管理局 (FDA) 和欧洲药物管理局 (EMA) 现在在批准药物时强调患者体验数据.
- 定性试验研究 (ITR) 为决策提供了宝贵的患者见解.
研究的目的:
- 评估定性试验研究 (ITR) 在监管提交和卫生技术评估 (HTA) 审查中的程度和影响.
- 确定将患者体验数据整合到药物开发中的挑战和机会.
主要方法:
- 从2017年1月到2025年3月对监管指导文件,药物批准记录 (FDA/EMA) 和HTA报告进行系统审查.
- 分析包括新药应用,特定指导系列 (例如,罕见疾病,瘤学),以及来自多个国家的HTA方法指导.
主要成果:
- 只有10个FDA和8个EMA批准的产品包含ITR,其中55%用于罕见疾病.
- ITR数据提供了有关症状,患者体验和有意义的治疗益处的见解.
- 很少有HTA机构对定性研究提供指导,其在报告中的包含是最小的.
结论:
- 尽管人们越来越认可,但ITR在监管和HTA过程中的使用是有限的.
- 早期的战略规划和与监管/HTA要求的协调对于最大限度地提高定性证据的价值至关重要.
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