Improving the Efficiency of Regulatory Decisions for Biosimilars and Interchangeable Biosimilars by Leveraging Real-World Data

利用真实世界数据提高生物仿制药和可互换生物仿制药监管决策的效率

• 批准号: 10618571
• 负责人: Catherine Marie Lockhart
• 金额: $ 131.14万
• 依托单位: ACADEMY OF MANAGED CARE PHARMACY, INC.
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-09-01 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10618571
• 关键词: Improving; Efficiency; Regulatory; Decisions; Biosimilars

Project Summary The digital sharing and storage of healthcare information—medical claims and electronic health records—has produced lakes of real-world data (RWD) in the modern health care landscape. This volume of digital data, along with revolutions in big-data analysis, provide a new opportunity to use RWD to produce real-world evidence (RWE). RWD/RWE can improve the regulatory science around the development and review of biosimilar drugs. RWD have the potential to capture information from tens of millions of patients in real- world clinical practice. The use of RWD/RWE to improve the efficiency of clinical trials for biosimilar products will speed the development of new biosimilars, the process of granting the regulatory designation of interchangeability, and encourage more rapid uptake of the products in the market. The lack of evidence on the quality of RWD and on the relevance of RWE for regulatory decision-making about biosimilars is a major obstacle to using big-data analyses of RWD/RWE for these decisions. The study, “Improving the Efficiency of Regulatory Decisions for Biosimilars and Interchangeable Biosimilars by Leveraging Real-World Data to Produce Real-World Evidence,” will provide the research community with analytical tools they can re-use for their own tests of interchangeability and other regulatory questions. In the proposed study, we will: Aim 1: Determine the quality of RWD and the relevance of RWE for regulatory decision- making. We will conduct a literature review and convene an expert panel to establish the data needs for regulatory approvals of new biosimilars and designations of interchangeability. Then we will determine whether and where RWD/RWE could reasonably be used to address regulatory data needs. Aim 2: Use RWD/RWE to emulate an FDA evaluation of interchangeability of a biosimilar drug. We will design and conduct a target trial emulation of a switching study and compare outcomes produced from the emulation to those obtained from the FDA’s evaluations of interchangeability of the reference drug.

项目摘要 医疗保健信息的数字共享和存储-医疗索赔和 电子健康记录在现代社会产生了大量的真实世界数据（RWD）， 医疗保健景观。这些数字数据的数量，沿着大数据的革命 分析，提供了一个新的机会，使用RWD产生真实世界的证据（RWE）。 RWD/RWE可以围绕以下内容的开发和审查改进监管科学： 生物类似药 RWD有潜力从真实的数千万患者中捕获信息- 世界临床实践。使用RWD/RWE提高临床试验效率 对于生物类似药产品将加速新的生物类似药的开发， 授予监管机构的可追溯性，并鼓励更快地 产品在市场上的占有率。 缺乏关于RWD质量和RWE监管相关性的证据 关于生物仿制药的决策是使用大数据分析的主要障碍。 RWD/RWE对这些决定负责。研究报告《提高监管效率 通过利用现实世界做出生物仿制药和可互换生物仿制药的决策 数据产生现实世界的证据，”将提供研究界， 分析工具，他们可以重复使用自己的测试的可扩展性和其他 监管问题。在建议的研究中，我们会： 目标1：确定RWD的质量和RWE与监管决策的相关性- 制作。我们将进行文献审查，并召集专家小组， 新生物类似药的监管批准和指定的数据需求 互换性.然后，我们将确定RWD/RWE是否以及在何处可以 合理地用于满足监管数据需求。 目的2：使用RWD/RWE模拟FDA对 生物类似药我们将设计并进行切换研究的目标试验仿真 并将仿真产生的结果与FDA的结果进行比较， 评价参比药物的可重复性。