Department of Health Services Research and Policy, Faculty of Public Health and Policy, London School of Hygiene and Tropical Medicin, London, UK
Centre for Cancer Biomarkers (CCBIO), University of Bergen, Bergen, Norway.
BMJ Open. 2024 Mar 14;14(3):e077297. doi: 10.1136/bmjopen-2023-077297.
This study aims to identify how real-world data (RWD) have been used in single technology appraisals (STAs) of cancer drugs by the National Institute for Health and Care Excellence (NICE).
Cross-sectional study of NICE technology appraisals of cancer drugs for which guidance was issued between January 2011 and December 2021 (n=229). The appraisals were reviewed following a published protocol to extract the data about the use of RWD. The use of RWD was analysed by reviewing the specific ways in which RWD were used and by identifying different patterns of use.
The number of appraisals where RWD are used in the economic modelling.
Most appraisals used RWD in their economic models. The parametric use of RWD was commonly made in the economic models (76% of the included appraisals), whereas non-parametric use was less common (41%). Despite widespread use of RWD, there was no dominant pattern of use. Three sources of RWD (registries, administrative data, chart reviews) were found across the three important parts of the economic model (choice of comparators, overall survival and volume of treatment).
NICE has had a long-standing interest in the use of RWD in STAs. A systematic review of oncology appraisals suggests that RWD have been widely used in diverse parts of the economic models. Between 2011 and 2021, parametric use was more commonly found in economic models than non-parametric use. Nonetheless, there was no clear pattern in the way these data were used. As each appraisal involves a different decision problem and the ability of RWD to provide the information required for the economic modelling varies, appraisals will continue to differ with respect to their use of RWD.
本研究旨在确定英国国家卫生与保健优化研究所（NICE）在癌症药物单技术评估（STA）中如何使用真实世界数据（RWD）。
对 2011 年 1 月至 2021 年 12 月发布指南的 NICE 癌症药物技术评估进行横断面研究（n=229）。根据已发表的方案，对评估进行审查以提取关于 RWD 使用的数据。通过审查 RWD 使用的具体方式和识别不同的使用模式来分析 RWD 的使用情况。
经济模型中使用 RWD 的评估数量。
大多数评估在其经济模型中使用 RWD。RWD 在经济模型中通常采用参数方法（纳入的评估中有 76%），而非参数方法则较少见（41%）。尽管广泛使用 RWD，但使用模式没有主导模式。在经济模型的三个重要部分（比较剂选择、总生存和治疗量）中发现了三种 RWD 来源（登记处、行政数据、图表审查）。
NICE 长期以来一直对 STA 中 RWD 的使用感兴趣。对肿瘤学评估的系统回顾表明，RWD 已广泛用于经济模型的不同部分。在 2011 年至 2021 年期间，经济模型中参数方法的使用比非参数方法更为常见。尽管如此，这些数据的使用方式并没有明确的模式。由于每个评估都涉及不同的决策问题，并且 RWD 提供经济建模所需信息的能力各不相同，因此评估将继续在其对 RWD 的使用方面存在差异。
