PaLaDIn

Developing new treatments for rare diseases is highly challenging. Because there are, by definition, very few patients with each rare disease, there is a major lack of data on patients’ needs, preferences and experiences of living with the disease. Furthermore, what little data exists is often fragmented and hard to access.

The aim of PaLaDIn is to address this head-on by developing a state-of-the-art platform dubbed the ‘Interactium’ to drive innovative, real-world data collection from patients with rare diseases. The project focuses on rare neuromuscular diseases (NMDs), specifically Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). The team plans to leverage the TREAT-NMD Global Registry Platform, which brings together over 60 NMD patient registries which collect patient data following a harmonised data model.

The Interactium will be able to integrate data from diverse sources, including patient-reported outcome/experience measures (PROMs and PREMs), as well as digital outcome measures from wearable devices, all of which will be co-created with patients.

A patient interface will facilitate the collection of data and information on preferences, and also allow users to visualise their data and control how it is used.

The project will maximise the utility, interoperability and reusability of the Interactium data so that it can be widely used by researchers and industry 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: on regulatory decision-making; monitoring patient care; creating standards of care; and facilitating clinical trials.

The project is coordinated by a patient organisation and the partners include experts in NMDs, patient advocacy and data science. They hope that their results will not only improve the lives of people with NMDs, but will prove useful to other rare disease communities around the world facing similar challenges.