英国联邦药品和医疗器械数据网络的监管视角:来自“研究马拉松”的经验教训。

IF 1.9 4区 医学 Q4 MEDICAL INFORMATICS
Helen P Booth, John Connelly, Daniel Dedman, Katherine Donegan, Alison Cave
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引用次数: 0

摘要

在国际上,医疗监管机构正在寻求更好地利用真实世界数据(RWD)来支持其决策。虽然英国国家卫生服务系统收集了全国人口从摇篮到坟墓的数据,但在不同护理环境中的数据孤岛化、互操作性和访问方面仍然存在挑战。2023年,举办了一场“Study-A-Thon”活动,探讨如何通过及时提高RWD的可用性,利用英国分布式数据网络的动员来生成用于监管目的的真实世界证据(RWE)。两个重点关注高优先级数据缺口(医疗设备和二级保健处方)的研究问题被选为案例研究来支持这项工作。本文总结了研究马拉松的细节,并讨论了英国药品和保健产品监管机构(MHRA)、英国利益相关者和国际合作伙伴在制定和实施RWD战略时反思的关键经验。讨论了数据的缺点,例如缺乏对患者在整个护理环境中的随访,以及需要开发通用数据模型以获取医疗产品使用情况的相关信息。从鼓励在护理点更好地收集数据到适当地解释结果,强调了当地数据和临床专业知识对成功的重要性。两项研究结果的成功交付支持了这样一种观点,即随着进一步的发展,英国联合数据模型可以增强整个产品生命周期的国家监管决策。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
A Regulatory Perspective on a UK Federated Data Network for Medicines and Medical Devices: Lessons from a 'Study-A-Thon'.

Internationally, medical regulators are seeking to make better use of real-world data (RWD) to support their decision making. While the UK National Health Service collects population-wide cradle-to-grave data, challenges remain around siloing, interoperability and access to data across different care settings. In 2023, a `Study-A-Thon' was held to explore how mobilisation of a UK distributed data network might be used to generate real-world evidence (RWE) for regulatory purposes by increasing availability of RWD in a timely manner. Two research questions focusing on high-priority data gaps (medical devices and secondary care prescribing) were selected as case studies to support this work. This paper summarises details of the Study-A-Thon and discusses key learnings for the UK's Medicines and Healthcare products Regulatory Agency (MHRA), UK stakeholders and international partners to reflect on when developing and implementing RWD strategies. Shortcomings of the data are discussed, such as a lack of follow-up for patients across care settings and the need to develop common data models to capture relevant information on medical product utilisation. The importance of local data and clinical expertise for success is highlighted, from encouraging better data collection at point of care through to appropriate interpretation of results. Successful delivery of results for both studies supports the view that, with further development, a UK federated data model could enhance national regulatory decision-making across the product lifecycle.

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来源期刊
Therapeutic innovation & regulatory science
Therapeutic innovation & regulatory science MEDICAL INFORMATICS-PHARMACOLOGY & PHARMACY
CiteScore
3.40
自引率
13.30%
发文量
127
期刊介绍: Therapeutic Innovation & Regulatory Science (TIRS) is the official scientific journal of DIA that strives to advance medical product discovery, development, regulation, and use through the publication of peer-reviewed original and review articles, commentaries, and letters to the editor across the spectrum of converting biomedical science into practical solutions to advance human health. The focus areas of the journal are as follows: Biostatistics Clinical Trials Product Development and Innovation Global Perspectives Policy Regulatory Science Product Safety Special Populations
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