PROMs are part of a triad of outcome measures, with the others being rater-based tests and objective tests. Rater-based tools include Expanded Disability Status Scale (EDSS) and Multiple Sclerosis Functional Composite (MSFC); however, the widely used EDSS has repeatedly been shown to have psychometric limitations, meaning that more responsive measures are needed. Sensitive measures are becoming increasingly important as growing numbers of patients with low disease activity are being treated, and so smaller differences need to be measured.1

PROMs can be used alongside biological and physical methods to measure treatment efficacy and disease course from the patients’ perspective.1,2 Examples of two classic PROMs used in clinical trials are the 29-item Multiple Sclerosis Impact scale (MSIS-29) and the 12-item MS Walking Scale (MSWS-12). The importance and scope of use of these measures is highlighted by their being translated into more than 75 languages.3 These and other PROMs can help to achieve high-quality patient-centred care; however, questions still remain regarding their accuracy and the context in which they should be used.2,4–6

“I believe there is a strong justification for advancing many of the patient-reported outcome instruments that we have”
– Prof. Jeremy Hobart

In this article, we present data from ECTRIMS 2019 on why PROMs are important and how PROMs research has progressed.

Why are patient reported outcome measures important?

PROMs are well established in MS clinical research, and expanding the use of PROMs into routine clinical care may facilitate uncovering aspects of MS that would otherwise go unrecognised. They may also allow clinicians to discover their patients’ priorities (particularly in terms of treatment goals), improve communication with their patients and implement shared decision-making.6

During a Hot Topics session at ECTRIMS 2019, data from a narrative review showed that there is a growing body of evidence that using patient-centred care models improve satisfaction with care and overall health outcomes. PROMs were seen to be central in this patient-centred approach as, despite the challenges around time limitations6 and interpretations, they provide valuable information on symptoms, treatment experiences, care preferences and daily living needs and values in a structured and consistent way over time.2,7 When patients were asked what they were looking for in a healthcare provider, one said:

“Open to your concerns, as opposed to trying to be a dictator telling you what you should be doing”
– Patient with MS discussing what they want from their MS care

Furthermore, Geremakis and colleagues reported on the impact of a patient-centred specialty model of care on PROMs. From these preliminary data collected via web-based PROMs, the group concluded that increasing patient access to care and quality of care at a MS patient-centred specialty practice improves patient experience with providers and their staff.7

Moving towards novel PROMs in MS

A study presented at ECTRIMS 2019 used the UK MS Registry (UKMSR), the largest repository of PROMs in the UK, to generate a new MS disability PROM. The UKMSR works with more than 40 NHS neurology clinics to collect and validate PROM data from patients with MS. In this study, 83 patients were assessed by EDSS and then by two PROMs: MSIS-29 v2 and the MSWS-12. When using multivariate linear regression the team was able to account for 94% of EDSS variance, meaning that these two PROMS were able to accurately predict EDSS outcomes in these patients with MS.8

For PROMs to be effectively used in clinical trials they must prove that they measure clearly defined concepts in specific clinical contexts. Further PROMs research presented at ECTRIMS aimed to develop a walking PROM that satisfied scientific and regulatory requirements for MS clinical trials. Using qualitative interviews of 59 patients with a variety of MS types and expert opinion, a conceptual framework of four primary domains around walking with MS was created. The group focused on activities related to walking and, using mixed methods, produced a 32-item PROM. In an independent cohort of 611 patients with MS, this novel PROM, named the MSWS-32, showed excellent performance characteristics and was conceptually and empirically superior to the currently used MSWS-12. Theoretically MSWS-32 represents a better primary endpoint measure for relapsing MS, secondary progressive MS and progressive MS in clinical trials.5

Conclusions

Currently, PROMs are being refined in clinical trials, but their use in clinical practice is in its relative infancy. Here we have highlighted some of the important reasons for PROMs to be used more extensively in clinical practice. Further research is needed to validate them and ensure their clinical utility. To this end, Prof. Hobart recommends a closer look at identifying concepts of interest, e.g. functions or symptoms and defining the context of use for each patient-focused outcome measure when creating PROMS, while Prof. Solari highlighted the need for these measures to be acceptable for use and meaningful for the patient and healthcare provider.3,6

References

  1. Schäffler N, Schönberg P, Stephan J, Stellmann JP, Gold SM, Heesen C. Comparison of patient-reported outcome measures in multiple sclerosis. Acta Neurol Scand 2013; 128: 114–21
  2. Finlayson M. PROMS: supporting patient-centred care. In: ECTRIMS. 2019: Hot Topic 9. 194
  3. J. Hobart. 20 years of MSIS-29 – importance of an MS specific QoL scale. In: ECTRIMS. 2019: Hot Topic 9. 193
  4. Nelson EC, Eftimovska E, Lind C, Hager A, Wasson JH, Lindblad S. Patient reported outcome measures in practice. BMJ 2015; 350: 1–3
  5. J. Hobart LB. Advancing walking measurement in multiple sclerosis clinical trials: a new patient-reported outcome measure. In: ECTRIMS. 2019: Scientific session 9. 216
  6. A. Solari. PROMs in standardized MS care. In: ECTRIMS. 2019: Hot Topic 9. 195
  7. C. Geremakis, D. Campagnolo, S. Stuart, A. Ahmad, N. Hu CT. The Impact an MS patient-centered specialty practice has on patient-reported outcomes related to their care team experience. In: ECTRIMS. 2019: P1184
  8. W. Rodgers, R. Middleton, H. Downing-Wood, K. Tuite-Dalton1, D.V. Ford RN. Towards a clinically validated PRO measurement of disability in people with MS. In: ECTRIMS. 2019: P451
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