Department of Health Services Research and Policy, Faculty of Public Health and Policy, London School of Hygiene and Tropical Medicine, London, UK
Centre for Cancer Biomarkers (CCBIO), University of Bergen, Bergen, Norway.
BMJ Open. 2022 Jan 6;12(1):e055985. doi: 10.1136/bmjopen-2021-055985.
Due to the limitations of relying on randomised controlled trials, the potential benefits of real-world data (RWD) in enriching evidence for health technology assessment (HTA) are highlighted. Despite increased interest in RWD, there is limited systematic research investigating how RWD have been used in HTA. The main purpose of this protocol is to extract relevant data from National Institute for Health and Care Excellence (NICE) appraisals in a transparent and reproducible manner in order to determine how NICE has incorporated a broader range of evidence in the appraisal of oncology medicines.
The appraisals issued between January 2011 and May 2021 are included following inclusion criteria. The data extraction tool newly developed for this research includes the critical components of economic evaluation. The information is extracted from identified appraisals in accordance with extraction rules. The data extraction tool will be validated by a second researcher independently. The extracted data will be analysed quantitatively to investigate to what extent RWD have been used in appraisals. This is the first protocol to enable data to be extracted comprehensively and systematically in order to review the use of RWD.
This study is approved by the Ethics Committee of the London School of Hygiene and Tropical Medicine on 14 November 2019 (17315). Results will be published in peer-reviewed journals.
由于依赖随机对照试验的局限性，真实世界数据（RWD）在丰富卫生技术评估（HTA）证据方面的潜在益处得到了强调。尽管人们对 RWD 的兴趣日益增加，但系统研究如何将 RWD 用于 HTA 的研究却很有限。本研究方案的主要目的是以透明和可重复的方式从英国国家卫生与保健优化研究所（NICE）的评估中提取相关数据，以确定 NICE 如何在肿瘤药物评估中纳入更广泛的证据。
本研究纳入了 2011 年 1 月至 2021 年 5 月期间发布的符合纳入标准的评估。新开发的数据提取工具包括经济评估的关键组成部分。将根据提取规则从已确定的评估中提取信息。该数据提取工具将由第二位研究人员进行独立验证。提取的数据将进行定量分析，以调查 RWD 在评估中被使用的程度。这是第一个能够全面系统地提取数据以审查 RWD 使用情况的方案。
本研究于 2019 年 11 月 14 日获得伦敦卫生与热带医学院伦理委员会的批准（17315）。研究结果将发表在同行评议的期刊上。
