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; 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针对儿科和孤儿药物的决策提供综合方法，以药物开发问题为中心。这将构成用于药物开发和评估的透明生态系统，该生态系统将支持采用建模和仿真（M＆S）方法及相关数据类型（包括真实单词数据，例如注册表和电子医疗保健数据）。 ERAMET的总体目的是提供并实施一个框架，以建立M＆S方法的可信度和相关结果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）用于数据和分析方法的高质量标准（包括M＆S和混合方法）的开发和验证。这些将涵盖计算M＆S，数字双胞胎，AI，混合方法，标准统计和药物计量学，作为分析方法以及替代数据类型以及诸如RWD，EHealth数据，注册表，历史监管提交，科学和（非）临床试验等替代数据类型和来源。 （3）一个基于AI的平台，该平台将自动化和优化数据收集，格式化，建模和仿真分析并实施信誉评估。作为ERAMET的一部分，生态系统将应用于五个用例，包括儿科外推和在4类稀有疾病中的药物益处/风险表征，即共济失调，依赖于输血的血红蛋白病，支气管肺发育不良和退化性神经肿瘤。计划通过EMA资格程序进行至少一种经过验证的M＆S工具的提交和监管批准。将提出培训，以使这种新方法熟悉监管评估者，药物开发人员和临床研究人员。