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

“细胞或基因疗法在治疗目前治疗无法治愈的患者方面显示出巨大的潜力。这些包括关节炎、肝病、几种类型的癌症和糖尿病溃疡。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在出院后的较长时间内。该项目将开发一个电子捕获系统，以评估患者接受这些新疗法时的症状和生活质量。患者将能够通过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将被标记给临床团队，以帮助促进患者安全。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的证据基础，以支持NHS的吸收。患者直接参与了项目的开发，并将为系统开发提供直接投入，以确保系统满足他们的需求。正在英国高级治疗中心推广的Datatrial Nucleus数据库平台将从多个来源收集数据，并将在评估这些治疗的成本效益方面发挥重要作用。该项目的领导者是Midlands &威尔士高级疗法治疗中心（MW-ATTC）-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合作的国家卓越中心。该项目借鉴了ATTC网络利益相关者的专业知识，伦敦高级治疗中心（ATC）网络，制药行业，包括ATMP开发商和生产商，如Chiesi和Autolus，细胞和基因治疗弹射器，英国监管机构和付款人。"

"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."