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Patient-reported outcomes assessment to support accelerated access to advanced cell and gene therapies: PROmics

患者报告的结果评估，以支持加速获得先进的细胞和基因疗法：PROmics

基本信息

批准号：
104777
负责人：
金额：
$ 166.74万
依托单位：
UNIVERSITY HOSPITALS BIRMINGHAM NHS FOUNDATION TRUST
依托单位国家：
英国
项目类别：
Collaborative R&D
财政年份：
2018
资助国家：
英国
起止时间：
2018 至无数据
项目状态：
已结题

来源：
https://gtr.ukri.org/projects?ref=104777
关键词：
Patient reported outcomes assessment support

项目摘要

"Cell or gene therapies show great potential in treating patients with conditions that cannot be cured with current treatments. These include arthritis, liver disease, several types of cancer, and diabetic ulcers. It is important to assess the impact of these new therapies on patient quality of life and symptoms, both at the point of receiving therapy and over the longer-term once out of hospital.The project will develop an electronic capture system to assess patient symptoms and quality of life when they receive these new therapies. Patients will be able to report how they feel on the treatment by using electronic devices. Any worrying symptoms will be flagged to the clinical team to help promote patient safety. These data will also be used to assess the effectiveness of treatment and be used as an evidence base for regulators and policy makers to support uptake in the NHS. Patients have been directly involved in development of the project and will provide direct input into the system development to ensure that it meets their needs.The Datatrial Nucleus database platform being rolled out across the Advanced Therapy Treatment Centres in the UK will collect data from multiple sources and will be important in allowing assessments of cost-effectiveness of these therapies.The project lead is the Midlands & Wales Advanced Therapy Treatment Centre (MW-ATTC) - a national centre of excellence in partnership with the Centre for Patient Reported Outcomes Research at The University of Birmingham, health technology companies Dignio and Datatrial. The project draws on expertise from stakeholders in the ATTC network, London's network of Advanced Therapy Centres (ATCs), the pharmaceutical industry including ATMP developers and producers such as Chiesi and Autolus, the Cell and Gene Therapy Catapult, UK regulators and payers."

“细胞或基因疗法在治疗目前治疗无法治愈的疾病方面显示出巨大的潜力。这些疾病包括关节炎、肝病、几种癌症和糖尿病溃疡。评估这些新疗法对患者生活质量和症状的影响非常重要，无论是在接受治疗时还是出院后的长期内。该项目将开发一种电子捕获系统，以评估患者在接受这些新疗法时的症状和生活质量。疗法。患者将能够使用电子设备报告他们对治疗的感受。任何令人担忧的症状都会被标记给临床团队，以帮助提高患者的安全。这些数据还将用于评估治疗的有效性，并用作监管机构和政策制定者支持 NHS 接受治疗的证据基础。患者直接参与了项目的开发，并将直接参与系统开发，以确保系统满足他们的需求。数据试验在英国的高级治疗中心推出的 Nucleus 数据库平台将从多个来源收集数据，对于评估这些疗法的成本效益非常重要。该项目的牵头者是中部和威尔士高级治疗中心 (MW-ATTC)，这是一个国家卓越中心，与伯明翰大学患者报告结果研究中心、健康技术公司 Dignio 和 DataTrial 合作。该项目绘制了 ATTC 网络、伦敦高级治疗中心 (ATC) 网络、制药行业（包括 Chiesi 和 Autolus 等 ATMP 开发商和生产商）、细胞和基因治疗弹射器、英国监管机构和付款人的专业知识。”