Work Package 5: Patient Journey
The objectives of Work Package 5 are to collect and utilise real world data to explore the ‘healthcare journey’ and define key events of people living with ALS across Europe. Integrating social determinants of health (SDOH) with clinical indicators develops a longitudinal and holistic view of the patient journey.
Patient Journey & Real-World data
While there remains an unmet need for an effective treatment for ALS, it is equally important to understand how best to capture the wellbeing of patients and caregivers as the disease progresses. We need to look at the often-subtle relationships between a condition of illness and the domains of a person’s life. and to determine the personal, societal and economic impact both of illness and interventions.
The healthcare journey is a sequence of events that usually begin when a person develops a need for care. The ‘journey’ encompasses touchpoints of experience. Understanding how people with ALS and their families navigate and experience the healthcare system is essential to maximising the potential of novel treatments and services. An ecological model to frames the illness journey, widens the research lens includes patients, caregivers and family.
Real world data (RWD) in the context of clinical health care are gathered outside of conventional randomised controlled clinical trials. These data may be generated through routine clinical practice, sources such as Electronic Health Records (EHR), observational studies, Disease Registries, medical devices and wearables. RWD are collected and processed in PRECISION ALS.
The data can be quantitative (numerical) or qualitative (text, images). Common data types include patient demographics, health behaviours, medical history, clinical outcomes (including patient-reported outcomes), patient experiences, resource use, costs, omics, laboratory measurements, imaging, free text (NICE real-world evidence framework 2022)
Social Determinants of Health (SDoH) are the non-medical, environmental conditions—where people are born, live, learn, work, play, and age—that heavily influence health outcomes and quality of life (WHO 2025).
WP5 of the PRECISION ALS project aims to enrich our collection of RWD with SDoH data, collected across the PRECISION study sites. This presents a wonderful opportunity for scientific investigation among a broader range of patients and expands the representative sample of experiences. In addition to Quality-of-Life measures, this enables Health Technology assessments, health economic modelling, and health utility analyses. While integrating SDoH into clinical practice facilitates personalised care, and effective care plans.
However, collecting such important – non-clinical – data can be challenging. To date, issues raised include discomfort with the nature of the social and economic questions, cultural expectations, concerns about what these data will be used for. We explore cultural differences in outcome measures across the PRECISION sites in Europe and the role of context in cross-national healthcare ecosystems.
WP5 maps the PRECISION data life cycle, data sources, and formatting at each PRECISION site, and monitors the quality of demographic, socio-economic and resource use data captured and processed on the patient data platform.
In parallel, a number of projects (industry sponsored) utilize extant real-world data to address some of the most pressing questions in ALS research field currently.
Leader: Dr Miriam Galvin
