Bridging the Gap: Using Foreign Real-World Data to Inform Interchangeable Biosimilar Approvals

项目摘要/摘要 这项研究将使用来自美国、意大利和丹麦的真实世界数据(RWD)来评估 满足可互换产品标准的替代方法(区域2.ii)。长的- 学期目标是为FDA制定使用国外RWD的建议，以改善 监管流程。这项研究将确定美国以外的RWD是否有可能 提高美国监管研究的威力和普适性。研究人员将使用一种 观察性/非干预性回顾队列设计，以解决两个具体目标 以下是方法： ·目标1：评价生物相似互换性的可行性和有效性(例如 Switch)研究使用来自美国的真实世界数据和来自外部的来源 美国的这项研究将评估来自两个外国来源的RWD的质量，并制定一项 用于协调数据的公共数据模型。首席调查员(PI)将设计一个 共享协议。合作研究人员将模拟一种生物相似物的切换研究 产品在美国、意大利和丹麦网站销售。PI将比较每个站点的结果和 将结果与现有的研究进行比较，以验证我们的发现。 ·目标2：就如何应对挑战向FDA提出建议 在其监管决策中使用来自美国以外的真实世界数据- 制造过程。根据目标1的经验教训，国际和平倡议将为 FDA需要考虑。指导意见将建议应对独特挑战的战略。 与收集、标准化和验证来自国际来源的RWD有关。 这项研究将展示使用国外RWD如何显著提高FDA的效率 监管过程，导致更多地采用安全有效的生物相似产品。这 创新的方法也可以作为在美国以外使用RWD的模式 跨不同治疗领域的监管决策。这项研究将有助于FDA 推进其向患者提供安全有效的药物的使命。

PROJECT SUMMARY/ABSTRACT This study will use real-world data (RWD) from the U.S., Italy, and Denmark to evaluate alternative approaches to meet the standard for interchangeable products (Area 2.ii.). The long- term objective is to develop recommendations for the FDA on using foreign RWD to improve regulatory processes. The study will determine the potential of RWD from outside the U.S. to improve the power and generalizability of U.S. regulatory studies. Researchers will use an observational/non-interventional retrospective cohort design to address two specific aims with the following methods: • Aim 1: Evaluate the feasibility and validity of a biosimilar interchangeability (e.g. switching) study using real-world data from the U.S. and sources from outside the U.S. The study will evaluate the quality of RWD from two foreign sources and develop a common data model to harmonize the data. The Principal Investigator (PI) will design a shared protocol. Co-Investigators will conduct emulations of a switching study of a biosimilar product at U.S., Italian, and Danish sites. The PI will compare the results at each site and compare the results with an existing study to validate our findings. • Aim 2: Develop recommendations for the FDA on how to address the challenges of using real-world data from outside the United States in its regulatory decision- making processes. Based on learnings from Aim 1, the PI will propose guidance for the FDA to consider. The guidance will recommend strategies to address the unique challenges associated with collecting, standardizing, and validating RWD from international sources. This study will show how using foreign RWD can significantly improve the efficiency of the FDA regulatory process, leading to increased adoption of safe and effective biosimilar products. This innovative approach could also serve as a model for using RWD from outside the U.S. in regulatory decision-making across different therapeutic areas. This study will help the FDA advance its mission of making safe and effective drugs available to patients.