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.

RFA-FD-20-030的关键路径研究所提案 抽象的 从历史上看，FDA将现实世界证据（RWE）作为其努力的关键组成部分 确保上市后的监视和医疗产品安全性评估。 21日 Century Cures Act要求FDA开发一个扩展RWD使用的框架 RWE支持已批准的药物的新迹象的批准。经过 利用通过医学的正常实践，安全信号和 可以在已经批准的疗法中确定新指示的功效。但是，如 大多数现有的RWD是在没有监管标准的情况下生成的， 存在挑战，以确保RWD适合使用，以生成能够告知的RWE 监管决策。使用现有RWD回答监管问题的努力可以识别 这些挑战和相应的解决方案，但是这项工作需要重大 数据和法规科学专业知识的数量，以充分优化RWD提取的使用 可操作的RWE。关键路径学院（C-Path）具有该数据专业知识，并将执行 一项试点项目，旨在促进RWD在新生儿中产生RWE，并进行学习 广泛适用于其他治疗区域。每年在美国，有10％的新生儿 天生的早产，迫切需要改善生存和结果。然而， 新药开发最少，大多数现有药物的证据不足 支持这种高风险人群的安全性，功效和剂量。 C-Path将利用其 数据协作中心流程和基础架构以开发RWD分析 平台。 RWD将通过与调查人员和数据合作访问 科学家。将实施特定的算法和机制以从中提取RWD 相关来源（汇总数据库和EMR），策划和标准化数据，以及 使分析能够生成可行的RWE。当数据远程分析时，C-Path将 验证处理工作流以提供质量保证。因此提取的数据将解决两个 新生儿种群中的关键未满足需求：1）当前缺乏可行的参考值 对于常规实验室测试（作为妊娠/产后年龄的函数）和2）缺乏疾病 支气管肺发育不良的进展模型。差距分析将确定关键课程 与访问，质量，提取，策划，标准化和访问，质量，提取，策划和 RWD的分析以及最佳生成可起作用的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。经验教训 然后可以将高风险人群推广到其他治疗区域。