Patient Lifestyle and Disease Data Interactium (PaLaDIn)

神经肌肉疾病是收集患者真实世界人口统计和临床相关数据的前沿。PaLaDIn是一个公私合作伙伴关系，将利用TREAT-NMD的全球注册平台。它使用患者数据来支持临床试验计划/招募以及更复杂的监管机构驱动的研究。利用其他合作伙伴在患者参与和FAIR数据方面的专业知识，这一公私合作伙伴关系将开发Interactium，这是一种新的综合患者数据方法。Interactium将使注册报告数据与患者报告的结果/体验措施以及其他数据（如可穿戴设备和物联网设备）保持一致，并共享整理后的整体数据，以改善结果和决策，并加速一系列利益相关者（患者、监管机构、行业和DARWIN等倡议）的创新。PaLaDIn的其他目标：•开发一个患者界面来收集患者偏好，以支持包容性方法，并允许数据可视化和控制其使用。•提供一流的工具，以支持整个神经肌肉领域的转化研究、护理和诊断决策的各个阶段，并制定标准操作程序，以允许在罕见疾病和更广泛的健康领域中使用。•在神经肌肉疾病领域之外提供交流和共享学习，以支持所有疾病的类似系统和能力的扩大开发。开发以患者为中心且具有成本效益的高级集成医疗保健解决方案需要明确定义的患者群体（数量庞大但可管理）、广泛的正在进行的研究和试验以及处于所有开发阶段的药物。神经肌肉疾病提供了所有这些；然而，互动机制和相关的进程和框架具有更广泛的潜力。一个能够从任何相关来源获取数据、在患者层面对数据进行调整并提供数据分析以支持患者、临床医生、监管机构和健康相关行业的决策的系统，同样适用于糖尿病等更常见的疾病。PaLaDIn关键交付成果•灵活的Interactium omni数据平台•改进用户体验的架构/模板•可穿戴基础设施的架构•标准化过程的新方法•罕见疾病患者组织的工具包。

Neuromuscular disease is at the forefront of collecting real-world demographic and clinically-relevant data from patients. PaLaDIn, a public private partnership, will leverage TREAT-NMD's Global Registry Platform. This uses patient data to support clinical trial planning/ recruitment as well as more complex regulator-driven studies. With other Partners’ expertise in patient engagement and FAIR data, this a public private partnership, will develop the Interactium, a new, integrated approach to patient data. The Interactium will align registry-reported data with Patient Reported Outcome/Experience Measures and other data e.g., wearables and Internet of Things devices, and share the collated, holistic data to improve outcomes and decision-making, and accelerate innovation for a range of stakeholders (patients, regulators, industry and initiatives like DARWIN). PaLaDIn’s other objectives: • Develop a patient interface to collect patient preferences to support an inclusive approach, and to allow visualization of data and control over its use. • Provide class-leading tools to support all stages of decision-making in translational research, care and diagnosis across the neuromuscular field and develop Standard Operating Procedures to allow utilization across the rare disease and wider health fields. • To provide communications and shared learning outside the neuromuscular disease field to support scale-up development of similar systems and capabilities across all diseases. Developing a patient-centric and cost-effective advanced integrated healthcare solution requires well defined patient groupsin significant, but manageable numbers, extensive ongoing research and trials and drugs at all stages of development. Neuromuscular diseases provides all of these; however, the Interactium and associated processes and frameworks have much wider potential. A system that can ingest data from any relevant source, align that data at patient level, and provide data analysis to support decision-making by patients, clinicians, regulators and health-related industries is equally relevant to more common diseases such as diabetes. PaLaDIn key deliverables • Flexible Interactium omni data platform • Architecture/template for an improved user experience • Architecture for wearable infrastructure • Novel methods for FAIRification process • Toolkit for rare disease patient organisations.