Department of Research and Development, Sanofi, Chilly-Mazarin, France.
Department of Research and Development, Sanofi, Chilly-Mazarin, France.
Clin Ther. 2020 May;42(5):926-938. doi: 10.1016/j.clinthera.2020.03.006. Epub 2020 Apr 25.
For this article, the authors compiled, summarized, and analyzed data from 27 cases in which real-world data (RWD) were applied in regulatory approval. The aims were to provide an overview of RWD, based on classifications per therapeutic area, age group, drivers of acceptability, utility, data sources, and timelines, and to present insights on how it has been applied in regulatory decision making to date.
Clarivate Analytics was commissioned to collect data from cases in which RWD was used for new drug applications and line extensions submitted to the European Medicines Agency (EMA), the US Food and Drug Administration (FDA), Health Canada, and Japan's Pharmaceuticals and Medical Devices Agency. The query resulted in 27 cases in which regulatory approval was associated with RWD. The data were then categorized and elaborated with supporting information gathered from public databases and company websites.
There were 17 identified cases in which RWD were used for new drug applications, and 10 for line extensions, between the years 1998 and 2019. Approvals were spread across regulatory bodies: the EMA alone (6 cases), the FDA alone (4 cases), or jointly between the EMA and FDA or other regulatory bodies. The applications were also distributed across age groups and therapeutic areas but were mostly applied in oncology and metabolism. The new drug applications of all 17 products were approved, with drugs from new drug applications initially marketed as orphan drugs. In most cases, RWD were used either as primary data, when noncomparative data were available to demonstrate tolerability and efficacy, or as supportive data when validating findings. Common sources of RWD have been health or medical records (16 cases) and registries (8 cases). Review timelines in which RWD were applied were than 1 year for new drug applications and between 3 and 10 months for line extensions.
The analysis of this study was limited in that the data were gathered from the commissioned query and may therefore have been nonexhaustive. Nonetheless, we recognize that the use of RWD has been gaining attention across the community and is expected to expand as a result of the various initiatives and efforts carried out in the sector. While the current application of RWD has been limited to specific cases, there is a potential to further explore and develop its application. Further refinements in the analytical processes, methodologies, and techniques would need to be established to achieve similar effects observed in randomized controlled trials.
本文作者编译、总结和分析了 27 例实际数据（RWD）应用于监管审批的案例中的数据。目的是基于治疗领域、年龄组、可接受性驱动因素、实用性、数据源和时间线的分类，提供 RWD 的概述，并介绍迄今为止它在监管决策中的应用方式。
Clarivate Analytics 受委托从 RWD 用于向欧洲药品管理局（EMA）、美国食品和药物管理局（FDA）、加拿大卫生部和日本药品和医疗器械管理局提交的新药申请和产品线扩展的案例中收集数据。查询结果为 27 例与 RWD 相关的监管批准案例。然后，使用来自公共数据库和公司网站收集的支持信息对数据进行分类和详细说明。
1998 年至 2019 年期间，有 17 例确定的 RWD 用于新药申请，10 例用于产品线扩展。批准的案例分布在不同的监管机构：EMA 单独批准（6 例），FDA 单独批准（4 例），或 EMA 和 FDA 或其他监管机构联合批准。申请也分布在不同的年龄组和治疗领域，但主要集中在肿瘤学和代谢领域。所有 17 种新药申请产品均获得批准，新药申请的药物最初被作为孤儿药上市。在大多数情况下，RWD 要么作为主要数据使用，当有非比较数据可证明耐受性和疗效时，要么作为支持性数据用于验证发现。RWD 的常见来源是健康或医疗记录（16 例）和登记处（8 例）。应用 RWD 的审查时间线对于新药申请超过 1 年，对于产品线扩展在 3 至 10 个月之间。
本研究的分析受到限制，因为数据是从委托查询中收集的，因此可能不完整。尽管如此，我们认识到，RWD 的使用已经引起了业界的关注，并预计随着该领域各项倡议和努力的开展，其使用将会扩大。虽然 RWD 的当前应用仅限于特定案例，但有可能进一步探索和开发其应用。需要建立更精细的分析过程、方法和技术，以实现随机对照试验中观察到的类似效果。
