A meeting of the global PROMS initiative – placing all people with MS at the centre
In November 2023, as members of the PROMS Engagement Coordination Team[1], we travelled from across the globe to Baveno, Italy, to discuss the important topic of how patient reported outcomes can help with “Bridging the gap between clinical research and care”.‘PROMS’ stands for Patient Reported Outcomes for MS. It’s a global initiative that aims to develop agreement on a set of standardised Patient Reported Outcomes (PROs) to be used in MS research and healthcare.
[1] Zaratin, P., Vermersch, P., Amato, M. P., Brichetto, G., Coetzee, T., Cutter, G., … & Groups, P. I. W. (2022). The agenda of the global patient reported outcomes for multiple sclerosis (PROMS) initiative: Progresses and open questions. Multiple Sclerosis and Related Disorders, 61, 103757.
PROs are increasingly being used across research and healthcare for all conditions – not just MS. We believe that it is important that people with lived experience are at the heart of developing and deciding on which PROs and PROMs are used, so that the outcomes of research are meaningful to those lived experiences.
Ultimately this will lead to better healthcare, better health outcomes, and improved quality of life, so we will be able to participate fully in society alongside the challenges we experience from living with MS.The global PROMS initiative aims to take a holistic approach, considering the integral relationship of all aspects of living with the condition, rather than focusing on clinical markers or single symptoms.
Patient reported outcomes are reports of a person’s health condition that comes directly from the person, based on his/her own perception. PROs allow an individual to share their lived experience about their health or quality of life. Examples include how they feel, what symptoms they are experiencing, or what they are able to do.
A patient-reported outcome measure (PROM) is a measure or tool that records and reports health-related data from patients – passively (for example through wearable devices) or actively, through a person inputting data about their experiences.
“Let’s not forget that people with MS are social and emotional beings and have a lot to offer in terms of love, laughter and friendship, if they are supported as much as possible to participate fully in society and live as well as possible alongside the condition.” Guy, ECT member
What did we learn from this global meeting?
This meeting was packed with ideas and discussion, but there were some messages that really stood out.
Knowing what you want to measure and how to measure it is crucial, as Professor Jeremy Hobart emphasised in his talk. “Unless we get the measurement right we will undermine all the work.”
To understand more about what we need to measure, our global survey aimed to capture the symptoms and experiences that have the biggest impact on daily life for people with MS, and how this changes relating to age, stage of disease, or even where people live in the world. Look out for the results of this survey later this year.
Many of the measures currently used in clinical trials haven’t changed in years. Dr Paul Kamudoni discussed the need for trials to focus less on relapses and more on how therapies affect progression. But how can we expect to capture and quantify important features such as silent or unrecognised progression, unless we develop better tools?
For example, the EDSS score might report one’s ability to walk, but that does not tell us how active one is at their EDSS stage, and whether or not one is engaged socially, with good quality of life, or whether one is wholly housebound. Bladder dysfunction can have a very large impact on people’s ability to participate in society, yet this is rarely measured and included as an outcome in trials. Dr Valerie Block described how measuring bladder function – such as through the DFree app – can provide more validity and better insight into disease progression.
To push forwards in this area, the MS community needs to strengthen the different networks we have for sharing data, and work alongside health authorities and regulators. If everyone is moving in the same direction, then industry will be incentivised to adopt consistent outcomes in clinical trials, which are measuring something meaningful for people with MS.
Improving the use of PROMs in research and clinical trials is just one aspect the global PROMS initiative is working on. Attendees at the meeting also discussed how we can better use PROMs in the clinic, to improve treatment decisions and care. One challenge is that even if we can measure changes in our experiences, and share these with our clinicians, what can our clinicians do or change that will make a difference to our lives? For example, how much would a certain ‘score’ on a PRO tool need to change by, to indicate that we needed to try a new DMT, or start a new symptomatic therapy? This will need to be a focus for future research.
“The true power of PROMs in MS care lies in their ability to personalise treatment and support. Each patient experiences the disease differently, with unique symptoms, challenges, and responses to treatments. By incorporating digital technology in PROMS, care plans can be tailored to meet the specific needs and aspirations of each individual. This patient-centred approach not only improves the quality of care but also enhances patient satisfaction and overall well-being.” Helga, ECT Co-Chair
Conclusion
Understanding what really needs to be measured, and getting the right tools to measure what’s most important, is essential—without this we don’t have anything. Our global survey for people with MS will bring fascinating insights into how the impact of MS varies across ages, stages of the condition, and across the world.
The big challenge for effective patient engagement is always to ensure representation of the patient community[1]. This is where the ECT has such an important role – we have the responsibility to help turn individual patient perspectives into a collective one, for example through our recent global survey. In consultation with the rest of the PROMS Initiative team, we chose the functional domains and their definitions for the survey. We hope that this helps other people with MS who are taking part in the survey understand what is being asked of them – and as a result we’ll have a larger and more representative engagement of the community. So far we have more than 5000 responses from all over the world! Thanks to our work in the ECT, the PROMS Initiative is able to ensure representativeness of people with MS, in line with the model that was developed by the European funded project MULTI-ACT[2]
Everyone’s experience of MS is complex and individual to them, but the survey data will help us know what types of symptoms or experiences are the most important to capture in research and healthcare. We’re also looking at how effective the available PRO measures/tools are at capturing what’s most important. If the global community can agree to use the same measures, then we’ll start collecting enough data to really test whether these measures are working—or whether we need to develop new ones. Alongside this, the PROMS initiative is looking at what types of digital technology is already being used to capture and share the experiences of people with MS, and how this could be improved and used more widely.
“If a non-invasive, personalised way of assessing our condition over time, not just in a 15-minute, biannual appointment, were available—and used by health care teams, with approval and reimbursement from payers—there is no burden too onerous to prevent us from using and engaging with it.” Angela, ECT member
[1] Zaratin, P., Khan, U., & Graffigna, G. (2023). Comment on “Reflections on patient engagement by patient partners: How it can go wrong”. Research Involvement and Engagement, 9(1), 1-4.
[2] Zaratin, P., Bertorello, D., Guglielmino, R., Devigili, D., Brichetto, G., Tageo, V., … & Di Luca, M. (2022). The MULTI-ACT model: the path forward for participatory and anticipatory governance in health research and care. Health Research Policy and Systems, 20(1), 22.