Advancing standards and methodologies to generate real world evidence from real world data through a neonatal pilot project

推进标准和方法，通过新生儿试点项目从现实世界数据生成现实世界证据

• 批准号: 10250393
• 负责人: Jonathan M. Davis
• 金额: $ 174.82万
• 依托单位: CRITICAL PATH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-09-01 至 2023-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10250393
• 关键词: Advancing; standards; methodologies; generate; real

Critical Path Institute Proposal for RFA-FD-20-030 ABSTRACT Historically, FDA has utilized real-world evidence (RWE) as a key component of its efforts to assure post-marketing surveillance and evaluation of medical product safety. The 21st Century Cures Act mandates FDA to develop a framework that expands the use of RWD and RWE to support approvals of a new indications for an already approved drug. By leveraging data generated through the normal practice of medicine, signals of safety and efficacy for new indications can be identified in already approved therapies. However, as most existing RWD was generated without the regulatory standards in mind, significant challenges exist to ensure RWD is fit-for-purpose to generate RWE capable of informing regulatory decisions. Efforts to use existing RWD to answer regulatory questions can identify these challenges and corresponding solutions, but this endeavor requires a significant amount of data and regulatory science expertise to fully optimize the use of RWD to extract actionable RWE. The Critical Path Institute (C-Path) has that data expertise and will execute a pilot project to facilitate the use of RWD to generate RWE in neonates, with learnings being broadly applicable to other therapeutic areas. Each year in the U.S., 10% of neonates are born preterm and there is an urgent need to improve survival and outcome. However, there is minimal new drug development and most existing drugs have insufficient evidence to support safety, efficacy, and dosage in this high-risk population. C-path will leverage its Data Collaboration Center processes and infrastructure to develop an RWD Analytics Platform. RWD will be accessed through collaborations with investigators and data scientists. Specific algorithms and mechanisms will be implemented to extract RWD from relevant sources (aggregated databases and EMRs), curate and standardize data, and enable analyses to generate actionable RWE. When data are analyzed remotely, C-Path will validate processing workflows for quality assurance. Data thus extracted will address two key unmet needs in the neonatal population: 1) current lack of actionable reference values for routine laboratory tests (as a function of gestational/postnatal age) and 2) lack of disease progression model for bronchopulmonary dysplasia. A gaps analysis will identify key lessons specific to neonates relating to the access, quality, extraction, curation, standardization, and analysis of RWD, and potential solutions to optimally generate actionable RWE identified, and shared with key stakeholders in the neonatal community. Lessons learned with this high-risk population can then be generalized to other therapeutic areas.

关键路径研究所针对 RFA-FD-20-030 的提案 抽象的 从历史上看，FDA 一直利用真实世界证据 (RWE) 作为其努力的关键组成部分 确保医疗产品安全性的上市后监督和评估。 21日 《世纪治愈法案》要求 FDA 制定一个扩大 RWD 使用范围的框架 和 RWE 支持批准已批准药物的新适应症。经过 利用正常医学实践产生的数据、安全信号和 可以在已经批准的疗法中确定新适应症的疗效。然而，作为 大多数现有的 RWD 是在没有考虑监管标准的情况下生成的，这很重要 确保 RWD 适合目的以生成能够提供信息的 RWE 存在挑战 监管决策。使用现有 RWD 来回答监管问题的努力可以确定 这些挑战和相应的解决方案，但这一努力需要大量 大量的数据和监管科学专业知识，以充分优化 RWD 的使用来提取 可操作的莱茵集团。关键路径研究所 (C-Path) 拥有数据专业知识并将执行 一个试点项目，旨在促进使用 RWD 在新生儿中产生 RWE，并吸取经验教训 广泛适用于其他治疗领域。在美国，每年有 10% 的新生儿 早产儿，迫切需要提高生存率和预后。然而， 新药开发很少，大多数现有药物证据不足 支持这一高危人群的安全性、有效性和剂量。 C-path 将利用其 用于开发 RWD 分析的数据协作中心流程和基础设施 平台。 RWD 将通过与调查人员和数据的合作来访问 科学家。将实施特定的算法和机制来提取RWD 相关来源（聚合数据库和电子病历），整理和标准化数据，以及 使分析能够生成可操作的 RWE。当远程分析数据时，C-Path 将 验证处理工作流程以保证质量。由此提取的数据将解决两个问题 新生儿群体中未满足的关键需求：1) 目前缺乏可操作的参考值 用于常规实验室检查（作为孕周/产后年龄的函数）和 2) 没有疾病 支气管肺发育不良的进展模型。差距分析将找出关键教训 针对新生儿的有关获取、质量、提取、管理、标准化和 RWD 分析，以及确定的最佳生成可操作 RWE 的潜在解决方案， 并与新生儿社区的主要利益相关者分享。从中吸取的教训 然后可以将高危人群推广到其他治疗领域。