Baumfeld Andre Elodie, Carrington Nate, Siami Flora S, Hiatt Jo Carol, McWilliams Carly, Hiller Carolyn, Surinach Andy, Zamorano Alejandro, Pashos Chris L, Schulz Wade L
Roche Diagnostics, Santa Clara, California, USA.
Roche Diagnostics, Indianapolis, Indiana, USA.
Clin Pharmacol Ther. 2022 Dec;112(6):1172-1182. doi: 10.1002/cpt.2565. Epub 2022 Apr 29.
Real-world data (RWD) and real-world evidence (RWE) are becoming essential tools for informing regulatory decision making in health care and offer an opportunity for all stakeholders in the healthcare ecosystem to evaluate medical products throughout their lifecycle. Although considerable interest has been given to regulatory decisions supported by RWE for treatment authorization, especially in rare diseases, less attention has been given to RWD/RWE related to in vitro diagnostic (IVD) products and clinical decision support systems (CDSS). This review examines current regulatory practices in relation to IVD product development and discusses the use of CDSS in assisting clinicians to retrieve, filter, and analyze patient data in support of complex decisions regarding diagnosis and treatment. The review then explores how utilizing RWD could augment regulatory body understanding of test performance, clinical outcomes, and benefit-risk profiles, and how RWD could be leveraged to augment CDSS and improve safety, quality, and efficiency of healthcare practices. Whereas we present examples of RWD assisting in the regulation of IVDs and CDSS, we also highlight key challenges within the current healthcare system which are impeding the potential of RWE to be fully realized. These challenges include issues such as data availability, reliability, accessibility, harmonization, and interoperability, often for reasons specific to diagnostics. Finally, we review ways that these challenges are actively being addressed and discuss how private-public collaborations and the implementation of standardized language and protocols are working toward producing more robust RWD and RWE to support regulatory decision making.
真实世界数据（RWD）和真实世界证据（RWE）正成为医疗保健领域监管决策的重要工具，并为医疗保健生态系统中的所有利益相关者提供了一个机会，以便在医疗产品的整个生命周期内对其进行评估。尽管RWE支持的治疗授权监管决策，尤其是在罕见病领域，已引起了广泛关注，但与体外诊断（IVD）产品和临床决策支持系统（CDSS）相关的RWD/RWE却较少受到关注。本综述审视了与IVD产品开发相关的当前监管实践，并讨论了CDSS在协助临床医生检索、筛选和分析患者数据以支持有关诊断和治疗的复杂决策方面的应用。该综述接着探讨了利用RWD如何能增强监管机构对检测性能、临床结果以及获益-风险概况的理解，以及如何利用RWD来增强CDSS并提高医疗实践的安全性、质量和效率。虽然我们列举了RWD协助IVD和CDSS监管的例子，但我们也强调了当前医疗系统中阻碍RWE潜力充分实现的确切挑战。这些挑战包括数据可用性、可靠性、可获取性、协调统一和互操作性等问题，这些问题通常是由于诊断方面的特定原因所致。最后，我们回顾了正在积极应对这些挑战的方式，并讨论了公私合作以及标准化语言和协议的实施如何致力于生成更可靠的RWD和RWE以支持监管决策。
