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

项目总结/摘要 这项研究将使用来自美国的真实世界数据（RWD），意大利和丹麦进行评估 满足可互换产品标准的替代方法（领域2.ii.）。很长的- 长期目标是为FDA制定关于使用国外RWD的建议，以改善 监管程序。该研究将确定美国以外的RWD的潜力， 提高美国监管研究的能力和普遍性。研究人员将使用 观察性/非干预性回顾性队列设计，以解决两个特定目标， 以下方法： ·目的1：评估生物类似药的可行性和有效性（例如， 切换）研究使用来自美国的真实世界数据和来自美国以外的来源 美国该研究将评估两种国外来源的RWD的质量， 统一的数据模型来协调数据。主要研究者（PI）将设计一个 共享协议共同研究者将模拟生物仿制药的转换研究 产品在美国，意大利和丹麦的网站。PI将比较每个研究中心的结果， 将结果与现有研究进行比较，以验证我们的发现。 目标2：为FDA制定关于如何应对挑战的建议 在其监管决策中使用来自美国以外的真实世界数据- 制作过程。根据目标1的学习，PI将为 FDA考虑。该指南将提出应对独特挑战的战略建议 与从国际来源收集、标准化和验证RWD相关。 本研究将展示使用外国RWD如何显著提高FDA的效率 监管过程，导致安全有效的生物类似药产品的采用增加。这 创新的方法也可以作为一个模式，使用RWD从美国以外的， 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.