对新型瘤药物的同情获取计划的分析
Sarah A Kelly1, Karine E Ronan2, Mohammed Zameer2
1School of Medicine, University College Dublin, Dublin, Ireland. Kellys50@tcd.ie.
富有同情心的获取计划 (CAPs) 为42%的晚期癌症患者提供了临床益处. 然而,许多患者由于治疗迟到而没有接受治疗,这凸显了及时批准药物的必要性.
科学领域:
- 在瘤学瘤学.
- 药物经济学 药物经济学
- 临床药理学 临床药理学
背景情况:
- 爱尔兰对新型瘤药物的有限补偿影响了患者的获取.
- 富有同情心的获取计划 (CAPs) 提供对有益但未完全批准的癌症药物进行管理的获取.
- 对于等待国家退款或药物全面授权的患者来说,CAPs至关重要.
研究的目的:
- 通过CAPs评估接受新型瘤药物的患者的临床益处和结果.
- 评估使用CAPs的患者的治疗持续时间和反应率.
- 确定与CAPs在晚期恶性瘤治疗中的挑战和局限性.
主要方法:
- 在Mater医院对113名晚期恶性瘤患者的回顾性评估 (2012年8月 - 2022年7月).
- 收集的数据包括疾病类型,接受的CAP治疗,持续时间和最佳反应.
- 临床益处定义为放射性完整,部分或稳定的疾病反应.
主要成果:
- 93名患者接受了CAP治疗,42% (47/113) 获得了临床益处.
- 7名患者实现了完全反应 (CR).
- 27% (30/113) 没有接受计划治疗或在3个月内死亡,这表明潜在的延迟.
结论:
- 很大一部分患者在十年内从CAP中获得了临床益处.
- 由于疾病进展或死亡率,迟到获得CAP仍然是一个挑战.
- CAP提供了有意义的患者利益,但不能取代新型癌症疗法的及时批准和退款.
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