Mofid Sarah, Bolislis Winona Rei, Kühler Thomas C
Global Regulatory Science and Policy, Sanofi R&D, Chilly-Mazarin, France.
Global Regulatory Science and Policy, Sanofi R&D, Chilly-Mazarin, France.
Clin Ther. 2022 Feb;44(2):306-322. doi: 10.1016/j.clinthera.2021.12.010. Epub 2022 Jan 21.
This article provides an analysis of the various regulatory decisions available in the public domain that suggest the use of real-world data (RWD) for postmarketing surveillance activities of products that have a marketing authorization approved by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). The study focuses on the cases in which RWD was used for postapproval commitments or requirements (and to a lesser extent label extensions, as this has been previously published) of medicinal products comprising small molecules and biologics to support efficacy claims or confirm an acceptable safety profile.
Clarivate Analytics was commissioned to collect data from cases in which RWD was used in the postapproval settings submitted to the EMA (data were found covering the last 14 years) and the FDA (data were found spanning 23 previous years). The query resulted in 165 cases in which regulatory approval was associated with RWD. The data were then categorized and expanded with supporting information gathered from public databases and company websites.
The use of RWD to support regulatory decision-making in the postmarketing surveillance setting has increased in recent years. Most postmarketing surveillance activities are legally enforced requests on the marketing authorization holder to further document the product's safety profile. Data drawn from registries tend to be the most common source in this regard.
RWD have increasingly been used in recent years, both for new product approvals and line extensions and, as shown in this study, in the postapproval setting. There is now a growing appreciation of the potential of RWD as a source in its own right to support regulatory decision-making on the benefits and risks associated with clinical interventions.
本文分析了公共领域中可用的各种监管决策，这些决策表明可将真实世界数据（RWD）用于已获得欧洲药品管理局（EMA）和美国食品药品监督管理局（FDA）批准上市产品的上市后监测活动。该研究聚焦于将RWD用于小分子和生物制品类药品的批准后承诺或要求（以及程度较低的标签扩展，因为此前已有相关发表），以支持疗效声明或确认可接受的安全性概况的案例。
委托科睿唯安公司从提交给EMA（发现的数据涵盖过去14年）和FDA（发现的数据跨越此前23年）的批准后使用RWD的案例中收集数据。查询结果得到165个监管批准与RWD相关的案例。然后对数据进行分类，并用从公共数据库和公司网站收集的支持信息进行扩充。
近年来，在上市后监测环境中使用RWD支持监管决策的情况有所增加。大多数上市后监测活动是对上市许可持有人的法定强制要求，以进一步记录产品的安全性概况。在这方面，来自注册登记处的数据往往是最常见的来源。
近年来，RWD越来越多地被用于新产品批准和产品线扩展，并且如本研究所示，也用于批准后环境。现在人们越来越认识到，RWD本身作为一种资源，在支持有关临床干预的益处和风险的监管决策方面具有潜力。
