医疗技术评估中药物的长期相对有效性的不确定性
Jan-Willem Versteeg1, Rick Vreman2, Aukje Mantel-Teeuwisse1
1Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
概括
卫生技术评估 (HTA) 机构在长期药物有效性方面的不确定性. 方法和证据的接受有很大差异,影响了患者的准入,需要定制的管理入口协议和重新评估.
科学领域:
- 卫生经济学 卫生经济学
- 制药政策 制药政策
- 基于证据的医学基于证据的医学.
背景情况:
- 药物的长期相对有效性的不确定性是医疗技术评估 (HTA) 的一个关键挑战.
- 医疗技术评估机构使用各种方法来解决评估中的这种不确定性.
研究的目的:
- 调查不同国家HTA机构如何解决关于药物的长期有效性的不确定性.
- 在各种治疗领域分析这些评估的方法和结果.
主要方法:
- 对来自6个国家HTA机构的49份HTA报告进行了系统审查.
- 报告评估了9种用于脊髓肌肉缩,囊性纤维化和高胆固醇血症的药物,包括81种相对有效性和45种成本效益评估.
- 收集了包括试验,结果,长期有效性不确定性,管理入口协议和重新评估的数据.
主要成果:
- 在91%的成本效益和74%的相对有效性评估中考虑了长期有效性不确定性.
- 由于方法和时间的不同,在纳入长期疗效数据方面观察到显著差异.
- 提出了23个管理入口协议 (14个与不确定性相关),并进行了13次重新评估,其中4次导致患者访问增加.
结论:
- 卫生技术评估机构面临着长期有效性不确定性的重大挑战,导致证据接受和决策的异质性.
- 管理入口协议和重新评估用于管理不确定性,但在HTA机构和指示中应用不一致.
- 在HTA机构之间证据接受的差异凸显了对标准化方法的需求,以最大限度地提高患者获得创新药物的机会.
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