WP1 focuses on the overall coordination and management of the PaLaDIn project. It ensures that the project progresses smoothly, stays on track, and achieves its objectives on time and within budget. This includes day-to-day management, monitoring progress, coordinating all partners, managing finances and contracts, and overseeing data management, regulatory compliance, and risk mitigation. WP1 also facilitates communication between partners and the European Commission to ensure efficient and transparent project implementation.

Objectives of the WP:

• Coordinate the implementation of the project according to the agreed work plan and Horizon Europe regulations.
• Monitor scientific, technical, and administrative progress, ensuring that milestones and deliverables are achieved on time.
• Facilitate communication and collaboration between partners, subcontractors, and the European Commission.
• Manage the project’s finances, including budget distribution, expense monitoring, and financial reporting.
• Ensure proper data management, regulatory compliance, and protection of intellectual property rights.

Potential benefits:

An effective project management structure will enable PaLaDIn to achieve its goals efficiently, fostering collaboration across all partners and ensuring high-quality outcomes.

Organisations involved:

All PaLaDIn consortium partners.

Objective(s)

1. Improve and grow the collection of PROM’s and PREM’s in the neuromuscular field
2. Provide patients with a user interface that can support their healthcare journey by providing an engaging experience in new and existing patients

The WP will develop education and support materials that ensure patients are as informed as possible about joining a registry, use of their data and the implications of using the research and information tools within the Interactium.

The purpose of WP3 is to communicate and widely disseminate project updates and results, both within the Europe and beyond, reaching key stakeholders including health care decision makers, clinicians, patients, registry curators and the wider general public. Our ultimate goal is to identify rare disease communities that could benefit from tools and resources developed within PaLaDIn and support the adoption of those resources through the development of toolkits.

Objectives of the WP:

• To ensure effective external communication is delivered by the consortium to all stakeholders.
• To maximise the impact by scaling up success from PaLaDIn firstly to all neuromuscular diseases and then across the wider rare disease field.
• To widely disseminate project results within the Europe and beyond, reaching health care decision makers, clinicians, patients, registry curators and the wider general public.

Potential benefits:

By communicating effectively about PaLaDIn with all stakeholders, we will support the adoption of resources and tools developed within PaLaDIn in the neuromuscular and wider rare disease field. Ultimately ensuring that patients are able to benefit from the knowledge and tools generated within the project.

Organisations involved:

Newcastle University, Parent Project, TREAT-NMD, FSHD Society

The purpose of this work package is to ensure that data from patient registries and patient-generated data in the Interactium data platform are optimally integrated, governed, and made FAIR (Findable, Accessible, Interoperable, and Reusable). This work package focuses on creating a data infrastructure that supports advanced decision-making for care, treatments, and research, while safeguarding patient privacy and consent.

Objectives

• To ensure that data collected within the Interactium can be accessed and reused (under clearly defined conditions) both by registries and patients contributing data, as well as by the broader research and healthcare community, so that it can guide decisions on care, treatments, and outcomes that matter to patients.
• To create strong guidelines for data (re)use, to ensure data is managed in line with patient consent, privacy laws such as the GDPR, and good clinical practice.

Potential benefits

• Accelerated and more reliable decision-making for clinicians, patients, and researchers through access to high-quality, interoperable data.
• Enhanced trust in data visiting or sharing by empowering patients with control over their data and consent.
• Improved data quality and readiness for AI and statistical analysis, supporting innovation in healthcare.
• Scalable methodologies and tools that can be adopted across rare and common disease fields.

Organisations involved

Leiden University Medical Center (LUMC), Amsterdam UMC, TREAT-NMD, Newcastle University, FSHD Society, 4MedBox (via LUMC)

WP5 is focused on driving the design and build of the Interactium^® – a global, patient-centric data platform, and will work with a subcontractor of TREAT-NMD to design and build the Interactium^®. The build of the platform will follow an Agile methodology, allowing for extensive stakeholder engagement throughout the project development, build and delivery; ensuring value is provided at every stage of the project. WP5 brings together the work and contributions from work packages within the project and stakeholders to combine expertise and shared learnings in the creation of the Interactium^® data platform.

Objectives of the WP:

• To create an open, transparent, privacy-focused patient-centric data management and support system.
• To build such a system on FAIR (Findable, Accessible, Interoperable, Reusable) Principles and make such data available to the medical and research community.
• To build such an architecture that any data could be ingested now and, in the future, to meet the needs of the growing demand for high-quality PROMs (Patient Reported Outcome Measures) and PREMs (Patient Reported Experience Measures) data.

Potential benefits:

Overall, the Interactium^® data platform will link together data collected in patient registries with data collected directly from patients, providing a comprehensive view and natural history of a patient’s condition and could be used to support healthcare decision-making by using data that are important to patients and reflects their lived experience.

Organisations involved:

TREAT-NMD, Amsterdam University Medical Centre, FSHD Society

Objective(s)

1. Work with patient groups to develop new digital outcome measures and explore the use of wearables.
2. Incorporate pose estimation analysis of video data to analyse motor function
3. Bring onboard a digital partner and a device manufacturer to support the development of the interoperability architecture.
4. To prove the benefit of patient-generated health data via video capture and the use of consumer grade and medical devices and use that real world data as part of research methodology.

WP6 will capture video data and wearable data from major consumer-and medical-grade devices, and develop the required interoperability architecture with the Interactium e-Health Platform.

Objective(s)
1. Publication of a harmonised consensus FSHD Dataset,
2. Publication of a harmonised consensus DM Dataset.
3. Development of a framework to govern future dataset updates.

WP7 will apply the successful consensus process developed by TREAT-NMD to the DM and FSHD datasets. The process involves forming a working group of experts in a particular disease and developing and agreeing on an initial list of data items. We aim to produce decipherable FSHD and DM datasets capable of supporting novel but also ongoing late-stage trials and post-authorisation studies in the future.

Objective(s)
1. Consolidation and validation of existing tools of used by the DMD Hub
2. Development of tools for empowering patients to become active participants in their care, to allow clinicians and patients to generate data-led research questions
3. Work with other PaLaDIn consortium partners to enable the adoption of the resulting DMD tools in other disease areas. This will be initiated through workshops to demonstrate utility of tools in DMD and how these can be translated to other rare diseases.
4. Demonstrate the impact of the tools developed for DMD:

All the tools will be the result of a co-creation process that will include stakeholder workshops (e.g. patients, clinicians, industry, HTA) to initiate, refine or finalise content, functionality and user-interface. Integrating the tools and services developed into the one cohesive Interactium platform will increase usability and therefore patient engagement. It will also avoid duplication of effort and resources (including patients’ time).

1. To develop, test and deploy frameworks that will enable rare-disease patients and patient advocacy organisations to lead, scale and harmonise cross-sector collaboration through utilising the Interactium.
2. To create a set of SOPs and a toolkit that can be applied by patient organisations working in any disease area.
3. To ensure patients are supported through decisions concerning participation in clinical trials.
4. To generate an Interactium-compatible HTA assessment model for FSHD with a set of SOPs to enable other groups to recreate this work.

In the rare disease space, collection of data from patients is often driven by patient advocacy organisations. As the importance and use of patient-derived data increases, these organisations will be able to play a greater role in bringing together collaborative efforts between academia, industry, and patients. TREAT-NMD plans to make the Interactium available to registries, patient organisations and other similar stakeholders.

WP9 will deploy an existing operating framework in use in Canada and the US (Project Mercury) that enables disease-specific patient organisations to lead collaboration among all stakeholders at the country and continent level. This framework will unify, orient, and organise groups to meet the needs of a specific rare disease community.

The WP will deploy a Clinical Trial Concierge (CTC) program which comprises highly trained lay Patient Liaisons (PL) who are members of registered patient advocacy organisations. PLs will support patients in their journey to find out more about clinical trials. They will also help research and healthcare staff understand more about the experiences of those who take part in research, thus supporting the development of PREMs.