Ecosystem for rapid adoption of modelling and simulation METhods to address regulatory needs in the development of orphan and paediatric medicines

快速采用建模和模拟方法的生态系统，以满足孤儿药和儿科药物开发中的监管需求

• 批准号: 10106221
• 金额: $ 74.78万
• 依托单位: UNIVERSITY OF WARWICK
• 依托单位国家: 英国
• 项目类别: EU-Funded
• 财政年份: 2024
• 资助国家: 英国
• 起止时间: 2024 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=10106221
• 关键词: Ecosystem; rapid; adoption; modelling; simulation

ERAMET will provide an integrated approach for developers and regulators’ decision-making for paediatric and orphan drugs, centred on the drug development questions. This will constitute a transparent ecosystem for drug development and assessment, that will facilitate the adoption of modelling and simulation (M&S) methods and related data types (including real word data such as registries and electronic healthcare data). The overall objective of ERAMET is to provide and implement a framework for establishing the credibility of M&S methods and related results as sources of evidence within regulatory procedures. The ecosystem proposed by ERAMET will be based on three pillars: (1) A repository connecting questions, data and methods. (2) The development and validation of high-quality standards for data and analytical methods (including M&S and hybrid approaches). These will cover computational M&S, digital twins, AI, hybrid approaches, standard statistics and pharmacometrics, as analytical methods and alternative data types and sources such as RWD, eHealth data, registries, historical regulatory submissions, scientific and (non)clinical trials). (3) An AI-based platform that will automate and optimise the data collection, formatting and modelling and simulation analysis and implement the credibility assessment. As part of ERAMET, the ecosystem will be applied to five use-cases including paediatric extrapolation and characterisation of drug benefit/risk in 4 groups of rare diseases, namely ataxia, transfusion dependent haemoglobinopathies, bronchopulmonary dysplasia, and degenerative neuromuscular. Each of the use-case is planned to lead to submission and regulatory approval of at least one validated M&S tool via the EMA qualification procedure. Training will be proposed to familiarise regulatory assessors, drug developers and clinical researchers with this new approach.

ERAMET 将为开发商和监管机构提供以药物开发问题为中心的儿科和孤儿药决策的综合方法，这将构成药物开发和评估的透明生态系统，从而促进建模和模拟（M&S）的采用。 ）方法和相关数据类型（包括登记和电子医疗数据等真实数据），ERAMET 的总体目标是提供和实施一个框架，以建立 M&S 方法和相关结果的可信度，作为监管程序中的证据来源。 ERAMET 提出的生态系统将基于三个支柱：（1）连接问题、数据和方法的存储库（2）数据和分析方法（包括 M&S 和混合方法）的高质量标准的开发和验证。涵盖计算 M&S、数字孪生、人工智能、混合方法、标准统计和药理学，作为分析方法和替代数据类型和来源，例如 RWD、电子健康数据、注册、历史监管提交、科学和（非）临床(3) 一个基于人工智能的平台，将自动化和优化数据收集、格式化、建模和模拟分析，并实施可信度评估。作为 ERAMET 的一部分，该生态系统将应用于五个用例，包括儿科外推和表征。 4 组罕见疾病的药物获益/风险，即共济失调、输血依赖性血红蛋白病、支气管肺发育不良和退行性神经肌肉病。计划通过 EMA 资格程序提交至少一种经过验证的 M&S 工具并获得监管机构批准，并将开展培训，以使监管评估人员、药物开发商和临床研究人员熟悉这一新方法。