Bullement Ash, Podkonjak Tanja, Robinson Mark J, Benson Eugene, Selby Ross, Hatswell Anthony J, Shields Gemma E
Delta Hat, Nottingham, UK.
Takeda UK Ltd, London, UK.
Int J Technol Assess Health Care. 2020 Jul 10:1-7. doi: 10.1017/S0266462320000434.
To establish how real-world evidence (RWE) has been used to inform single technology appraisals (STAs) of cancer drugs conducted by the National Institute for Health and Care Excellence (NICE).
STAs published by NICE from April 2011 to October 2018 that evaluated cancer treatments were reviewed. Information regarding the use of RWE to directly inform the company-submitted cost-effectiveness analysis was extracted and categorized by topic. Summary statistics were used to describe emergent themes, and a narrative summary was provided for key case studies.
Materials for a total of 113 relevant STAs were identified and analyzed, of which nearly all (96 percent) included some form of RWE within the company-submitted cost-effectiveness analysis. The most common categories of RWE use concerned the health-related quality of life of patients (71 percent), costs (46 percent), and medical resource utilization (40 percent). While sources of RWE were routinely criticized as part of the appraisal process, we identified only two cases where the use of RWE was overtly rejected; hence, in the majority of cases, RWE was accepted in cancer drug submissions to NICE.
RWE has been used extensively in cancer submissions to NICE. Key criticisms of RWE in submissions to NICE are seldom regarding the use of RWE in general; instead, these are typically concerned with specific data sources and the applicability of these to the decision problem. Within an appropriate context, RWE constitutes an extremely valuable source of information to inform decision making; yet the development of best practice guidelines may improve current reporting standards.
确定真实世界证据（RWE）如何被用于为英国国家卫生与临床优化研究所（NICE）开展的癌症药物单一技术评估（STA）提供信息。
对NICE在2011年4月至2018年10月期间发布的评估癌症治疗的STA进行审查。提取有关使用RWE直接为公司提交的成本效益分析提供信息的情况，并按主题进行分类。使用汇总统计数据来描述出现的主题，并为关键案例研究提供叙述性总结。
共识别并分析了113份相关STA的材料，其中几乎所有（96%）在公司提交的成本效益分析中都包含某种形式的RWE。RWE使用最常见的类别涉及患者的健康相关生活质量（71%）、成本（46%）和医疗资源利用（40%）。虽然RWE的来源在评估过程中经常受到批评，但我们仅发现两例RWE的使用被明确拒绝的情况；因此，在大多数情况下，向NICE提交的癌症药物申请中RWE被接受。
RWE已在向NICE提交的癌症申请中被广泛使用。向NICE提交的申请中对RWE的主要批评很少是关于RWE的总体使用；相反，这些批评通常涉及特定的数据来源以及这些来源对决策问题的适用性。在适当的背景下，RWE是为决策提供信息的极其有价值的信息来源；然而，制定最佳实践指南可能会提高当前的报告标准。
