Sidney Kimmel Comprehensive Cancer Center, Division of Hematologic Malignancies, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Sidney Kimmel Comprehensive Cancer Center, Division of Hematologic Malignancies, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Clin Ther. 2019 Feb;41(2):336-349. doi: 10.1016/j.clinthera.2018.12.013. Epub 2019 Jan 30.
In light of recently published guidelines from the US Food and Drug Administration (FDA) on the communication of real-world data (RWD) and real-world evidence (RWE) to support regulatory decision making, it is important to understand how such data are developed, the limitations of these data, and how to best use RWD to improve patient care. Historically, the use of RWE has been approached with skepticism because of its often-retrospective nature compared with data from conventional randomized controlled trials (RCTs). This review discusses the role and function of RWE and RWD in clinical research. We summarize the types of RWE used in clinical research, outline the challenges and limitations involved with these data, and suggest how these types of analyses can supplement results from clinical trials to foster a more complete understanding of a drug or disease area of interest. In particular, we focus on the role of RWE in investigating chronic myeloid leukemia (CML) and tyrosine kinase inhibitor therapy for CML.
We reviewed FDA guidance on the use of RWE and conducted a PubMed literature search to evaluate published data from real-world studies in CML.
RWE includes analysis of RWD gathered from nonconventional sources, including patient registries, observational studies, and social media, among others. Importantly, although real-world studies do not adhere to the same degree of controlled conditions and predefined patient-management strategies as do conventional clinical trials, analyses resulting from these studies can be held to a high degree of validation and standardization, making them as meaningful as those from RCTs. In CML, RWE has informed early treatment milestones and has provided a window into patient perspectives regarding treatment. These types of analyses have already informed and can continue to inform disease management. These improvements in disease management, in turn, will help clinicians to better forecast treatment challenges and allow for the optimization of future treatment paradigms.
Real-world studies are different from conventional RCTs and therefore provide insight into distinct aspects of treatment and patient outcomes. Together with results from clinical trials, RWE can help to illustrate a more complete picture of the tolerability, effectiveness, and impact of a drug. The recently published guidelines indicate that the FDA expects a growing role for RWE.
鉴于美国食品和药物管理局（FDA）最近发布的关于使用真实世界数据（RWD）和真实世界证据（RWE）来支持监管决策的指南，了解这些数据的开发方式、这些数据的局限性以及如何最好地使用 RWD 来改善患者护理非常重要。历史上，由于与传统随机对照试验（RCT）相比，RWE 通常具有回顾性，因此人们对其使用持怀疑态度。本综述讨论了 RWE 和 RWD 在临床研究中的作用和功能。我们总结了用于临床研究的 RWE 类型，概述了这些数据所涉及的挑战和局限性，并提出了如何利用这些类型的分析来补充临床试验结果，以更全面地了解药物或疾病领域。特别是，我们重点关注 RWE 在研究慢性髓性白血病（CML）和 CML 的酪氨酸激酶抑制剂治疗中的作用。
我们回顾了 FDA 关于使用 RWE 的指南，并进行了 PubMed 文献检索，以评估 CML 真实世界研究中的已发表数据。
RWE 包括对非传统来源（包括患者登记处、观察性研究和社交媒体等）收集的 RWD 的分析。重要的是，尽管真实世界研究不像传统临床试验那样遵守相同程度的受控条件和预设的患者管理策略，但这些研究产生的分析可以达到高度的验证和标准化，使其与 RCT 一样有意义。在 CML 中，RWE 为早期治疗里程碑提供了信息，并为患者对治疗的看法提供了一个窗口。这些类型的分析已经提供了信息，并且可以继续为疾病管理提供信息。这些疾病管理的改进反过来将帮助临床医生更好地预测治疗挑战，并为优化未来的治疗模式提供依据。
真实世界的研究与传统的 RCT 不同，因此可以深入了解治疗和患者结局的不同方面。与临床试验结果相结合，RWE 可以帮助更全面地说明药物的耐受性、有效性和影响。最近发布的指南表明，FDA 预计 RWE 将发挥越来越大的作用。
