Penberthy Lynne T, Rivera Donna R, Lund Jennifer L, Bruno Melissa A, Meyer Anne-Marie
Surveillance Research Program, Division of Cancer Control and Population Sciences, National Cancer Institute, Rockville, Maryland.
Department of Epidemiology, Gillings School of Global Public Health, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.
CA Cancer J Clin. 2022 May;72(3):287-300. doi: 10.3322/caac.21714. Epub 2021 Dec 29.
Generating evidence on the use, effectiveness, and safety of new cancer therapies is a priority for researchers, health care providers, payers, and regulators given the rapid pace of change in cancer diagnosis and treatments. The use of real-world data (RWD) is integral to understanding the utilization patterns and outcomes of these new treatments among patients with cancer who are treated in clinical practice and community settings. An initial step in the use of RWD is careful study design to assess the suitability of an RWD source. This pivotal process can be guided by using a conceptual model that encourages predesign conceptualization. The primary types of RWD included are electronic health records, administrative claims data, cancer registries, and specialty data providers and networks. Careful consideration of each data type is necessary because they are collected for a specific purpose, capturing a set of data elements within a certain population for that purpose, and they vary by population coverage and longitudinality. In this review, the authors provide a high-level assessment of the strengths and limitations of each data category to inform data source selection appropriate to the study question. Overall, the development and accessibility of RWD sources for cancer research are rapidly increasing, and the use of these data requires careful consideration of composition and utility to assess important questions in understanding the use and effectiveness of new therapies.
鉴于癌症诊断和治疗的快速变化，为研究人员、医疗服务提供者、支付方和监管机构提供有关新型癌症治疗方法的使用、有效性和安全性的证据是当务之急。使用真实世界数据（RWD）对于了解在临床实践和社区环境中接受治疗的癌症患者中这些新治疗方法的使用模式和结果至关重要。使用RWD的第一步是进行仔细的研究设计，以评估RWD来源的适用性。这个关键过程可以通过使用鼓励预先设计概念化的概念模型来指导。所纳入的RWD的主要类型包括电子健康记录、行政索赔数据、癌症登记处以及专业数据提供者和网络。必须仔细考虑每种数据类型，因为它们是为特定目的而收集的，为此在特定人群中捕获一组数据元素，并且它们在人群覆盖范围和纵向性方面存在差异。在本综述中，作者对每个数据类别的优势和局限性进行了高层次评估，以为适合研究问题的数据源选择提供参考。总体而言，用于癌症研究的RWD来源的开发和可及性正在迅速增加，使用这些数据需要仔细考虑其构成和效用，以评估在理解新疗法的使用和有效性方面的重要问题。
