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. You can learn more about the Global PROMS Initiative within the sections of our website.


[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 Disorders61, 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.

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.

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[2]. 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. [3]

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. 

Do you want to keep up to date with the latest news from the PROMS initiative? You can find us on LinkedInTwitter and YouTube.


[2] Zaratin, P., Khan, U., & Graffigna, G. (2023). Comment on “Reflections on patient engagement by patient partners: How it can go wrong”. Research Involvement and Engagement9(1), 1-4.

[3] 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 Systems20(1), 22.

Watch PROMS Plenary Event 2023 sessions on your YouTube channel:

Session 1: Role of PROMs and patient-generated data in unmasking ‘unrecognized’ progression

Session 2: Valuing the experiential knowledge of people living with the disease

Session 3: Lessons Learnt from existing PROMs

Session 4: The opportunity of digital PRO measurement: myth or reality?

Session 5: Building on the outcomes of the PROMS Plenary event: the Regulatory Agency’s perspective