Periodic Reporting for period 1 - PaLaDIn (Patient Lifestyle and Disease Data Interactium)
Reporting period: 2024-01-01 to 2024-12-31
Developing new treatments for rare diseases is highly challenging, due to limited numbers of patients to recruit for clinical trials, and a major lack of data on patients’ needs, preferences and experiences. Existing data is often fragmented and hard to access.
The PaLaDIn vision is to develop an innovative data collection platform (The Interactium) to accelerate the development of effective treatments and patient care, enhance healthcare decision-making and improve patient outcomes. The project will initially focus on real world data collection in rare neuromuscular diseases (NMDs), specifically Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD) and Myotonic Dystrophy (DM).
Project objectives include:
• Developing a patient interface to gather patient preferences for inclusivity and data visualization and control.
• Providing decision-making tools in translational research, care and diagnosis across the neuromuscular field and Standard Operating Procedures applicable across the rare disease and wider health fields.
• Facilitating communications and shared learning beyond neuromuscular diseases to develop similar systems and capabilities across all rare diseases.
The Interactium will integrate data from diverse sources, including patient registries, patient-reported outcome and experience measures (PROMs and PREMs), and wearable devices. It will be designed in co-creation with patients through patient advocacy organisations (namely Parent Project Aps, Duchenne UK and the FSHD Society).
The project will maximise the utility, interoperability and reusability of the Interactium data so that it can be widely used, with appropriate consents and permissions in place, by researchers, industry and deciscion makers working to improve the lives of people living with NMDs. In this way, the Interactium will incorporate the voices of people with NMDs in research, drug development and care planning.
The project will test the feasibility of using data from the Interactium to influence health research and care via four use cases:
• Patient data to support regulatory decision-making
• Using patient data to monitor patient care
• Creating standards of care guidelines using patient data
• Facilitate successful clinical trials
The project is coordinated by a patient organisation and the partners include experts in NMDs, patient advocacy and data science.
The PaLaDIn vision is to develop an innovative data collection platform (The Interactium) to accelerate the development of effective treatments and patient care, enhance healthcare decision-making and improve patient outcomes. The project will initially focus on real world data collection in rare neuromuscular diseases (NMDs), specifically Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD) and Myotonic Dystrophy (DM).
Project objectives include:
• Developing a patient interface to gather patient preferences for inclusivity and data visualization and control.
• Providing decision-making tools in translational research, care and diagnosis across the neuromuscular field and Standard Operating Procedures applicable across the rare disease and wider health fields.
• Facilitating communications and shared learning beyond neuromuscular diseases to develop similar systems and capabilities across all rare diseases.
The Interactium will integrate data from diverse sources, including patient registries, patient-reported outcome and experience measures (PROMs and PREMs), and wearable devices. It will be designed in co-creation with patients through patient advocacy organisations (namely Parent Project Aps, Duchenne UK and the FSHD Society).
The project will maximise the utility, interoperability and reusability of the Interactium data so that it can be widely used, with appropriate consents and permissions in place, by researchers, industry and deciscion makers working to improve the lives of people living with NMDs. In this way, the Interactium will incorporate the voices of people with NMDs in research, drug development and care planning.
The project will test the feasibility of using data from the Interactium to influence health research and care via four use cases:
• Patient data to support regulatory decision-making
• Using patient data to monitor patient care
• Creating standards of care guidelines using patient data
• Facilitate successful clinical trials
The project is coordinated by a patient organisation and the partners include experts in NMDs, patient advocacy and data science.
Two key workshops were held to define the Interactium platform’s architecture and user permissions. These discussions refined user journeys and engagement strategies with registry curators. A repository of potential data sources was also curated to support future integrations, along with a detailed development plan for the Interactium.
The project is developing a patient-centered interface for intuitive data visualization and user-friendly data sharing. A cross-work package working group was formed to align patient organizations’ efforts in dashboards and interfaces development, promoting knowledge sharing and efficiency. Multilingual surveys have gathered input from patient and caregiver to inform design. Ongoing research focuses on innovative data visualization and control mechanisms, like patient lockers, with future engagement planned with the TREAT-NMD registry network.
The project established a Data Management Plan, a Data Governance Framework and Committee and conducted stakeholder analysis to identify key actors involved in treatment evaluation and approval. Collaboration with HTA bodies and regulators aims to define best practices for regulatory decision-making. Plans are in progress to ensure the long-term sustainability and business viability of the Interactium platform.
Early steps have also been taken in developing tools for translational research, care, and diagnosis, including a technical specification for clinical trial access tools, and efforts to expand Project Mercury (a global iniatitve to speed up the development of medicines for FSHD) across the EU.
The project is developing a patient-centered interface for intuitive data visualization and user-friendly data sharing. A cross-work package working group was formed to align patient organizations’ efforts in dashboards and interfaces development, promoting knowledge sharing and efficiency. Multilingual surveys have gathered input from patient and caregiver to inform design. Ongoing research focuses on innovative data visualization and control mechanisms, like patient lockers, with future engagement planned with the TREAT-NMD registry network.
The project established a Data Management Plan, a Data Governance Framework and Committee and conducted stakeholder analysis to identify key actors involved in treatment evaluation and approval. Collaboration with HTA bodies and regulators aims to define best practices for regulatory decision-making. Plans are in progress to ensure the long-term sustainability and business viability of the Interactium platform.
Early steps have also been taken in developing tools for translational research, care, and diagnosis, including a technical specification for clinical trial access tools, and efforts to expand Project Mercury (a global iniatitve to speed up the development of medicines for FSHD) across the EU.
PaLaDIn's early developments are paving the way for a substantial impact in scientific, economic, and societal area.
Scientific Impact
The project is fostering collaboration between patient organizations working in different neuromuscular disease areas, promoting knowledge sharing and innovation. Early-stage efforts on new global datasets for FSHD and DM1 will enhance scientific research by establishing internationally agreed standards. These datasets, combined with the project’s commitment to FAIR (Findable, Accessible, Interoperable, Reusable) principles, will improve data integration and research capabilities. Additional innovations include the development of Patient-Reported Experience Measures (PREMs) and patient lockers, for better data visualization and control.
Economic Impact
By defining new datasets, the project aims to boost research in underrepresented disease areas, and attract pharmaceutical investment. The development of tools for clinical trial recruitment and accessibility will improve trial efficiency, lower costs and improve accessibility for rare disease treatments, effectively tackling the critical challenge of patient recruitment.
Societal Impact
PaLaDIn is patient-centric, aiming to improve access to care and treatment for individuals with rare neuromuscular diseases. With only 5% of rare diseases currently having approved therapies, the project engages patients, healthcare providers, and regulators to raise awareness and promote equitable healthcare solutions.
To maximize impact, further efforts are required in:
• research & demonstration: Ongoing engagement with researchers, regulators, and patient communities is essential for refining methodologies that address real-world needs.
• Regulatory frameworks: Collaboration with health authorities will help integrate project outputs into regulatory processes, ensuring widespread adoption.
• Sustainability & market access: Business planning will ensure the long-term sustainability for the Interactium platform post-project. , ensuring its continued availability and utility beyond the project’s duration.
Scientific Impact
The project is fostering collaboration between patient organizations working in different neuromuscular disease areas, promoting knowledge sharing and innovation. Early-stage efforts on new global datasets for FSHD and DM1 will enhance scientific research by establishing internationally agreed standards. These datasets, combined with the project’s commitment to FAIR (Findable, Accessible, Interoperable, Reusable) principles, will improve data integration and research capabilities. Additional innovations include the development of Patient-Reported Experience Measures (PREMs) and patient lockers, for better data visualization and control.
Economic Impact
By defining new datasets, the project aims to boost research in underrepresented disease areas, and attract pharmaceutical investment. The development of tools for clinical trial recruitment and accessibility will improve trial efficiency, lower costs and improve accessibility for rare disease treatments, effectively tackling the critical challenge of patient recruitment.
Societal Impact
PaLaDIn is patient-centric, aiming to improve access to care and treatment for individuals with rare neuromuscular diseases. With only 5% of rare diseases currently having approved therapies, the project engages patients, healthcare providers, and regulators to raise awareness and promote equitable healthcare solutions.
To maximize impact, further efforts are required in:
• research & demonstration: Ongoing engagement with researchers, regulators, and patient communities is essential for refining methodologies that address real-world needs.
• Regulatory frameworks: Collaboration with health authorities will help integrate project outputs into regulatory processes, ensuring widespread adoption.
• Sustainability & market access: Business planning will ensure the long-term sustainability for the Interactium platform post-project. , ensuring its continued availability and utility beyond the project’s duration.