Designing Well-Being Intervention Trials for People With Progressive Multiple Sclerosis: The Importance of Understanding Usual Care Comparators

For people with progressive forms of multiple sclerosis (MS), there are few disease-modifying treatments available,^1,2 leaving much of MS care focused on the management of symptoms and cumulative disability. Research indicates that pharmaceutical approaches to symptom management can reduce the frequency and severity of symptoms and delay progression^3,4 and rehabilitation and self-management approaches can help people with progressive MS reduce the impact of symptoms,^5,6 restore some lost abilities,^6,7 and manage everyday life despite disability.^8,9 Approaches tested through research are often applied in clinical practice under the assumption that they will improve the overall well-being of people with progressive MS, even if available evidence has not included this subgroup of the MS population or explicitly reported this broader outcome.

Well-being (or wellness) is an individual’s capacity to lead a life that is purpose filled and engaged and embraces their full potential, including physical, emotional, spiritual, intellectual, social, environmental, and vocational functioning.¹⁰ Therefore, well-being is not just the absence of symptoms or the ability to perform daily activities but also about thriving in a balanced, meaningful, and fulfilling life. This implies that interventions focused on well-being require a holistic, multidimensional approach and intervention targets that go beyond specific symptoms, physiological parameters, or behaviors.

The importance of broader targets for MS care is apparent in the results of a recent international survey¹¹ that found more than 40% of people with MS reported worsening of their ability to perform activities of daily living during the previous 24 months and that more than 60% reported negative changes in physical functioning. Furthermore, respondents indicated that these negative changes were associated with factors beyond symptoms (eg, emotional and social factors, future outlook, emotional well-being, coping, self-esteem, and relationships with friends and family).

This brief background supports the need for a novel approach to address the well-being of people with MS, particularly those with progressive MS, whose symptoms and their impact may be particularly burdensome and/or intractable.¹² It is this need that drives our work.

We are an international multidisciplinary consortium of MS researchers and clinicians from 8 countries who are developing and evaluating a well-being intervention for people with progressive MS.¹³ One of the challenges we faced while designing our study was in understanding the similarities and differences in usual care across our settings and what that means for evaluating our intervention. The intent of this editorial is to share our insights regarding the usual care challenge.

The Usual Care Challenge

To evaluate our intervention, we are planning a comparative trial with the exact design still under consideration. Comparative studies, including randomized controlled trials, rely on comparisons between the experimental condition (intervention to be tested) and a control or comparator condition. In MS rehabilitation trials focused on symptom management, the comparator is often described as usual, routine, or standard care, suggesting that participants randomly assigned to this trial arm receive what is typically or routinely provided to patients in that clinic or region. Although published treatment guidelines can encourage the harmonization of comparator trial arms in medical trials, the commitment in rehabilitation to patient-centered, goal-directed, and tailored interventions can make such harmonization challenging. Standardized comparators may be more challenging for well-being interventions because the ways in which individuals achieve a meaningful, purpose-filled, and engaged life vary socioculturally and individually.^14,15 Further, usual care can vary widely regionally and nationally, making the generalization of trial findings difficult without details about usual care comparators.

Notably, the practice characteristics of rehabilitation and the breadth of interventions addressing well-being are both consistent with the definition of a complex intervention. Complex interventions include several interacting components, require engagement with multiple behaviors by both the provider and the recipient, have multiple targets and outcomes, and offer a degree of delivery flexibility. Thus, a single intervention can vary considerably depending on the recipient, their goals, the provider’s implementation of the tailoring process, and the resources made available by the health care system, health insurance, or individual.^16-18 Complexity is further affected by service-level factors and by an individual’s clinical characteristics.^19-21

Given these multiple factors, what constitutes usual care to support and promote well-being for people with progressive MS is largely unknown. This gap has also been identified in stroke rehabilitation.²⁰ However, in situations where a trial uses a usual care comparator or when the experimental intervention is added to usual care, a good understanding of what constitutes usual care is needed to accurately interpret results and their relevance to a particular setting. Although the importance of describing a usual care comparator is recognized in the context of trial reporting,^21,22 we argue that understanding and documenting usual care also need to be part of trial planning and implementation. Gathering information about usual care in potential study sites will facilitate decision-making about which sites to use, enhance understanding about the overlap between the intervention to be tested and usual care at each site, guide thinking about how to maximize generalizability, and support future implementation if the trial is successful. Indeed, a methodological review summarizing the current approaches to determining the components of usual care comparators highlighted the importance of researchers understanding the context in which a trial is going to be implemented.²³

Moving Forward

As part of our consortium’s work,¹³ we aimed to describe usual care to support improved well-being among people with progressive MS across sites in Norway, the United Kingdom, the United States, Canada, Australia, Belgium, Italy, and Denmark. We developed a brief survey for this purpose, with the goal of using the results to inform trial design and planning. We did not intend to conduct formal comparisons across countries but wanted to understand what services and resources were available to support well-being (eg, disciplines and programs available, service funding models, referral requirements, care coordination) and what this might mean for our trial comparators. We also wanted to know about the modes of service delivery available, the extent of the use of digital technologies, and the outcomes being monitored. We received ethical approval of our survey from the Queen’s University Health Sciences and Affiliated Teaching Hospitals Research Ethics Board (Canada; No. 6041507). An invitation to participate that included a link to the online survey was sent to all members of our study team, who then purposefully distributed it to colleagues who might be invited to operate a study site for our trial in the future.

In total, 50 individuals responded, with half from the UK. We also had responses from Italy, Australia, Denmark, Belgium, Canada, and Norway. Respondents were primarily neurologists (41%) and nurses (23%), with others being physiotherapists (14%), occupational therapists (12%), psychologists (2%), continence specialists (2%), or other profession (5%). Most (50%) worked in an academic hospital, with tertiary care settings and community-based hospitals or practices being the next most common. The vast majority (85%) reported that their workplace was an MS specialty center. Although 98% considered themselves to be MS specialists within their discipline, one-third of respondents reported serving fewer than 100 people with progressive MS annually and 16% reported serving more than 1000 annually.

Despite responses from different countries, 90% of respondents indicated that traditional health care services were publicly funded, likely due to the preponderance of European respondents. In comparison, less than one-third of respondents reported that well-being services were publicly funded. Instead, a mixed model that included both public and private funding sources was most common, followed by private pay (eg, private insurance or out-of-pocket payments). Of concern is that 56% reported that less than 25% of their patients with progressive MS have private health insurance to access services that support well-being.

Respondents were asked about the sources of the referrals to their service, and they could choose more than 1 option. Primary care physicians were the most common referral source (56%), followed by general (ie, non-MS) neurologists (50%) and then other health care providers 40%). Just over 25% of respondents also reported receiving self-referrals from people with MS. Given our respondents’ settings, it is not surprising that the majority (47%) reported that nurses were the team members most likely to coordinate the connections between people with progressive MS and services or resources to support well-being. Only 3% had a case manager or coordinator who offered such coordination services for people with progressive MS.

The proportion of time respondents spent delivering services to support the well-being of people with progressive MS was limited: The majority (58%) reported that they spent 25% or less of their time addressing well-being. The availability of other disciplines, services, or resources to address well-being was also limited; for example, fewer than 25% of respondents indicated that their center offered exercise programs for people with physical disabilities and a smaller proportion had a social worker (18%) or offered mental health programs (14%), spiritual support (12%), or assistive technology services (8%). Service providers such as physiotherapists, occupational therapists, dietitians, speech therapists, continence advisers, and psychologists were sometimes available through a center or through external agencies, where referrals were often required. Availability of digital health care technologies (DHCTs) to support well-being was not common, except for telehealth technologies, which were available to 56% of respondents. All other DHCTs were available to fewer than one-third of respondents. Even when available, DHCTs were integrated into care 40% or less of the time.

Main Considerations

Our survey results uncovered several issues that we must consider as we develop our intervention to improve well-being for people with progressive MS. Attending to these issues may be useful to others interested in supporting the well-being of people with progressive MS and may improve future research.

Contents of Services Still Unknown

We asked about what services were available, primarily focusing on the person/discipline or service (eg, nurse, social worker, support group). We cannot assume that the people providing services are focused on well-being, consistent with the broad definition provided earlier. However, based on what we found, service provision focused on well-being appears to be relatively sparse for people with progressive MS. This may be due to funding structures, time available during appointments, or other service factors. We are left wondering the extent to which well-being services reach people with progressive MS and whether any subgroups (eg, racially or ethnically minoritized individuals) are particularly disadvantaged given extant literature.^24-26

Gaps in Coordination of Well-Being Care

Care to support well-being is often coordinated by nurses in the centers where our respondents worked. Nevertheless, we cannot assume that the availability of nurses is ubiquitous.²⁷ Even when nurses are available, they may not have time to address well-being given the ever-increasing pressures they are under to provide support for disease-modifying treatments and other medical issues. Overall, our findings suggest that focusing on well-being issues represents a small fraction of most neurologists’ and nurses’ clinical time. Although case coordinators or link workers have become a useful resource in other areas of health care, and social prescribing²⁸ and patient navigators²⁹ are used to complement traditional health care professionals’ roles, very few MS centers have such personnel or offer these models of care provision.

Well-Being Supports Beyond Health Care

We need to consider whether the types of supports and services needed to address well-being—we thought of this as thriving in a balanced, meaningful, and fulfilling life—are aligned with traditional health care settings, which primarily operate in a medical model of care. If we think broadly about usual care for well-being, we need to recognize that care can be obtained from services that are not commonly linked to health care (eg, recreation, leisure, and social programs) and from informal systems of support. These aspects of care can be invisible if they are not consistently recorded in patient records or during clinical trials. Our survey captured only aspects of usual care that were provided in the context of the clinical setting. We need to capture other aspects of well-being support when describing usual care comparator arms.

Availability and Use of DHCTs

Despite growth in the use of DHCTs in neurological trials,³⁰ our findings indicated limited use of such technologies in clinical care. We need a better understanding of the factors contributing to this implementation gap so that evidence-based DHCTs can be considered as an adjuvant or alternative option to traditional in-person services.^31-34 Given technological improvements and increased technological literacy, providing patientswithchoicesin accessing and receiving services that include evidence-based DHCTs is worth considering. The use of DHCTs is particularly relevant if we want to provide accessible well-being services in the community.

Importance of Describing Usual Care

Finally, in a trial, we can often control aspects such as access to the intervention, the method and timing of its delivery, and the training of personnel providing it. As our survey results revealed, usual care to support the well-being of people with progressive MS varies widely. This highlights the need for a consistent way to collect data and report on the components of usual care (see the definition of complex interventions previously) so that we fully understand the comparator arm of our well-being trials.

Conclusion and Implications

Our consortium is committed to developing and evaluating a program to support people with progressive MS to live well and thrive in daily life. Our small survey of clinicians at potential study sites has helped inform our thinking and planning. Although our approach and sample size have limitations, our findings highlighted variability in available services, care team composition, and access to well-being support. Understanding these variations is a critical step to design interventions that are feasible, able to be implemented at scale, and centered on the diverse needs of people with progressive MS. For a clinical audience, our team’s efforts highlight that usual care looks different across clinics, regions, and countries, which highlights the importance of interpreting the results of trials with usual care comparators carefully. Our work also highlights the potential value of monitoring and addressing gaps in the provision and coordination of well-being–focused services in routine care.