The Multiple Sclerosis Care Unit.

Treatment of multiple sclerosis (MS) has become increasingly multifaceted and comprises not only a variety of disease-modifying drugs with different mechanism of action but also a wide range of symptomatic therapies. Today, it is not possible for the family physician or even many general neurologists to master the current treatment algorithm, and this calls for the establishment of multidisciplinary MS Care Units. The core of the MS Care Unit would, in addition to MS neurologists and nurses, typically comprise neuropsychologists, clinical psychologists, physiotherapists, occupational therapists and secretaries, and will work together with a group of different specialists on formalized diagnostic workup procedures, protocols for initiation and follow-up of disease-modifying therapies. It is obvious that the terms of performance of different MS Care Units will vary across regions and need to be balanced with clinical practice according to local conditions. Although the main objective for establishment of MS Care Units will be to offer the single MS patient seamless and correct management of the disease to increase patient satisfaction and quality of life, it may even be cost-effective for the society by maintaining the working ability and reducing the costs of home help and custodial care by keeping people with MS resourceful.

Multidisciplinary data infrastructures in multiple sclerosis: Why they are needed and can be done!

Personalized treatment is highly desirable in multiple sclerosis (MS). We believe that multidisciplinary measurements including clinical, functional and patient-reported outcome measures in combination with extensive patient profiling can enhance personalized treatment and rehabilitation strategies. We elaborate on four reasons behind this statement: (1) MS disease activity and progression are complex and multidimensional concepts in nature and thereby defy a one-size-fits-all description, (2) functioning, progression, treatment, and rehabilitation effects are interdependent and should be investigated together, (3) personalized healthcare is based on the dynamics of system biology and on technology that confirms a patient's fundamental biology and (4) inclusion of patient-reported outcome measures can facilitate patient-relevant healthcare. We discuss currently available multidisciplinary MS data initiatives and introduce joint actions to further increase the overall success. With this topical review, we hope to drive the MS community to invest in expanding towards more multidisciplinary and longitudinal data collection.

ECTRIMS/EAN Guideline on the pharmacological treatment of people with multiple sclerosis.

BACKGROUND: Multiple sclerosis (MS) is a complex disease with new drugs becoming available in the past years. There is a need for a reference tool compiling current data to aid professionals in treatment decisions. OBJECTIVES: To develop an evidence-based clinical practice guideline for the pharmacological treatment of people with MS. METHODS: This guideline has been developed using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) methodology and following the updated EAN recommendations. Clinical questions were formulated in Patients-Intervention-Comparator-Outcome (PICO) format and outcomes were prioritized. The quality of evidence was rated into four categories according to the risk of bias. The recommendations with assigned strength (strong and weak) were formulated based on the quality of evidence and the risk-benefit balance. Consensus between the panelists was reached by use of the modified nominal group technique. RESULTS: A total of 10 questions were agreed, encompassing treatment efficacy, response criteria, strategies to address suboptimal response and safety concerns and treatment strategies in MS and pregnancy. The guideline takes into account all disease-modifying drugs approved by the European Medicine Agency (EMA) at the time of publication. A total of 21 recommendations were agreed by the guideline working group after three rounds of consensus. CONCLUSION: The present guideline will enable homogeneity of treatment decisions across Europe.

The patient's perspective: How to create awareness for improving access to care and treatment of MS patients?

BACKGROUND: There is currently no known cure for multiple sclerosis (MS). Four stakeholders play a major role in MS: healthcare professionals, regulators, payers and patients. OBJECTIVE: In Europe, patients are represented by the European Multiple Sclerosis Platform (EMSP), which aims to improve MS management and patients' quality of life. RESULTS: The EMSP has recently shown that there are major disparities in Europe in terms of access to care and treatment. Implementing the Code of Good Practice and a standardised MS nurse training may be useful in harmonising MS management across Europe. Additionally, the burden for novel therapeutic options to be approved by regulatory agencies has to decrease in order to provide faster access of treatment to patients. Data collection (e.g. national registers) also appears crucial to help research and shape the most effective policy in each country. Finally, people with MS should get appropriate (financial) support in order to complete their studies and find a job, as their active participation in society requires proper access to education and employment. Moreover, as they are the ones affected by MS, they seem to be best placed to represent themselves and their needs and should be consulted more often during decision-making processes by policy makers, regulators and payers.

Multiple sclerosis registries in Europe - results of a systematic survey.

BACKGROUND: Identification of MS registries and databases that are currently in use in Europe as well as a detailed knowledge of their content and structure is important in order to facilitate comprehensive analysis and comparison of data. METHODS: National MS registries or databases were identified by literature search, from the results of the MS Barometer 2011 and by asking 33 national MS societies. A standardized questionnaire was developed and sent to the registries' leaders, followed by telephone interviews with them. RESULTS: Twenty registries were identified, with 13 completing the questionnaire and seven being interviewed by telephone. These registries differed widely for objectives, structure, collected data, and for patients and centres included. Despite this heterogeneity, common objectives of the registries were epidemiology (n=10), long-term therapy outcome (n=8), healthcare research (n=9) and support/basis for clinical trials (n=8). While physician-based outcome measures (EDSS) are used in all registries, data from patients' perspectives were only collected in six registries. CONCLUSIONS: The detailed information on a large number of national MS registries in Europe is a prerequisite to facilitating harmonized integration of existing data from MS registries and databases, as well as comprehensive analyses and comparison across European populations.

Comparison of employment among people with Multiple Sclerosis across Europe.

Background: People with Multiple Sclerosis (PwMS) suffer from an increased risk of unemployment during the course of the disease. In recent years progress has been made in increasing the time until patients have to leave the workforce permanently. Such a retirement is often associated with MS but the driving factors including disability progression, support measures at the workplace, and societal aspects are not yet fully understood. Methods: We consolidated data from four European MS databases from Germany, Poland, Sweden, and the United Kingdom, which were able to provide data on working status, disability progression and quality of life in accordance with the data harmonization framework of the EUReMS (European Registry in Multiple Sclerosis) project. Results: Factors strongly associated with unemployment are disability progression, low quality of life and being close to the statutory retirement age. Overall, highest employment rate (77%) and lowest effects of gender and disease duration were found in Sweden. Conclusions: We found remarkable differences between the European registers and the countries studied, which may indicate inequalities at European level. Furthermore, our findings suggest that it is feasible and useful to combine data from different MS registers in Europe, albeit the data structures are heterogeneous.

Multiple Sclerosis Data Alliance - A global multi-stakeholder collaboration to scale-up real world data research.

The Multiple Sclerosis Data Alliance (MSDA), a global multi-stakeholder collaboration, is working to accelerate research insights for innovative care and treatment for people with multiple sclerosis (MS) through better use of real-world data (RWD). Despite the increasing reliance on RWD, challenges and limitations complicate the generation, collection, and use of these data. MSDA aims to tackle sociological and technical challenges arising with scaling up RWD, specifically focused on MS data. MSDA envisions a patient-centred data ecosystem in which all stakeholders contribute and use big data to co-create the innovations needed to advance timely treatment and care of people with MS.

Unmet needs, burden of treatment, and patient engagement in multiple sclerosis: A combined perspective from the MS in the 21st Century Steering Group.

BACKGROUND: Patient engagement is vital in multiple sclerosis (MS) in order to optimise outcomes for patients, society and healthcare systems. It is essential to involve all stakeholders in potential solutions, working in a multidisciplinary way to ensure that people with MS (PwMS) are included in shared decision-making and disease management. To start this process, a collaborative, open environment between PwMS and healthcare professionals (HCPs) is required so that similarities and disparities in the perception of key areas in patient care and unmet needs can be identified. With this patient-centred approach in mind, in 2016 the MS in the 21st Century Steering Group formed a unique collaboration to include PwMS in the Steering Group to provide a platform for the patient voice. METHODS: The MS in the 21st Century initiative set out to foster engagement through a series of open-forum joint workshops. The aims of these workshops were: to identify similarities and disparities in the perception and prioritisation in three key areas (unmet needs, the treatment burden in MS, and factors that impact patient engagement), and to provide practical advice on how the gaps in perception and understanding in these key areas could be bridged. RESULTS: Combined practical advice and direction are provided here as eight actions: 1. Improve communication to raise the quality of HCP-patient interaction and optimise the limited time available for consultations. 2. Heighten the awareness of 'hidden' disease symptoms and how these can be managed. 3. Improve the dialogue surrounding the benefit versus risk issues of therapies to help patients become fully informed and active participants in their healthcare decisions. 4. Provide accurate, lucid information in an easily accessible format from reliable sources. 5. Encourage HCPs and multidisciplinary teams to acquire and share new knowledge and information among their teams and with PwMS. 6. Foster greater understanding and awareness of challenges faced by PwMS and HCPs in treating MS. 7. Collaborate to develop local education, communication and patient-engagement initiatives. 8. Motivate PwMS to become advocates for self-management in MS care. CONCLUSION: Our study of PwMS and HCPs in the MS in the 21st Century initiative has highlighted eight practical actions. These actions identify how differences and gaps in unmet needs, treatment burden, and patient engagement between PwMS and HCPs can be bridged to improve MS disease management. Of particular interest now are patient-centred educational resources that can be used during time-limited consultations to enhance understanding of disease and improve communication. Actively bridging these gaps in a joint approach enables PwMS to take part in shared decision-making; with improved communication and reliable information, patients can make informed decisions with their HCPs, as part of their own personalised disease management.

What makes a compliant Phase III and pre-launch patient advocacy strategy?

BACKGROUND: A key task for the pharmaceutical industry is to understand the compliance implications of engaging with a patient advocacy group (PAG). This presents challenges for the industry to negotiate the ethical and reputational issues that can arise when working with a PAG. OBJECTIVE: To gain the views of pharmaceutical industry executives on future compliance challenges when working with PAGs. STUDY DESIGN: We conducted two surveys among two sets of industry executives: one group focussed on market access roles and the other focussed on non-market access roles. RESULTS: Transparency was identified as the biggest challenge, followed by project rationale and then by project ownership. CONCLUSION: We explore how this can be overcome and make recommendations on how best to work compliantly with PAGs.

Brain health: time matters in multiple sclerosis.

INTRODUCTION: We present international consensus recommendations for improving diagnosis, management and treatment access in multiple sclerosis (MS). Our vision is that these will be used widely among those committed to creating a better future for people with MS and their families. METHODS: Structured discussions and literature searches conducted in 2015 examined the personal and economic impact of MS, current practice in diagnosis, treatment and management, definitions of disease activity and barriers to accessing disease-modifying therapies (DMTs). RESULTS: Delays often occur before a person with symptoms suggestive of MS sees a neurologist. Campaigns to raise awareness of MS are needed, as are initiatives to improve access to MS healthcare professionals and services. We recommend a clear treatment goal: to maximize neurological reserve, cognitive function and physical function by reducing disease activity. Treatment should start early, with DMT and lifestyle measures. All parameters that predict relapses and disability progression should be included in the definition of disease activity and monitored regularly when practical. On suboptimal control of disease activity, switching to a DMT with a different mechanism of action should be considered. A shared decision-making process that embodies dialogue and considers all appropriate DMTs should be implemented. Monitoring data should be recorded formally in registries to generate real-world evidence. In many jurisdictions, access to DMTs is limited. To improve treatment access the relevant bodies should consider all costs to all parties when conducting economic evaluations and encourage the continuing investigation, development and use of cost-effective therapeutic strategies and alternative financing models. CONCLUSIONS: The consensus findings of an international author group recommend a therapeutic strategy based on proactive monitoring and shared decision-making in MS. Early diagnosis and improved treatment access are also key components.

Pooling real-world multiple sclerosis patient data on a European level: a true story of success.

Patient-based evidence is becoming increasingly important in budget-restricted healthcare systems as the information can help influence the direction of funds towards interventions that provide the most relevant outcomes for people living with a chronic disease. The European Multiple Sclerosis Platform (EMSP) is an umbrella organization for national multiple sclerosis (MS) patients' associations in Europe which represents the interests of >700,000 MS patients, their families and caregivers. EMSP aims to ensure the delivery of high-quality equitable treatment for persons with MS across Europe. EMSP is involved in numerous projects and activities that encompass its vision and mission. The European Network of MS Registries project has provided proof of concept that high quality MS data from previously unconnected sources can be integrated to inform research and improve patient outcomes.

Achieving patient engagement in multiple sclerosis: A perspective from the multiple sclerosis in the 21st Century Steering Group.

While advances in medicine, technology and healthcare services offer promises of longevity and improved quality of life (QoL), there is also increasing reliance on a patient׳s skills and motivation to optimize all the benefits available. Patient engagement in their own healthcare has been described as the 'blockbuster drug of the century'. In multiple sclerosis (MS), patient engagement is vital if outcomes for the patient, society and healthcare systems are to be optimized. The MS in the 21st Century Steering Group devised a set of themes that require action with regard to patient engagement in MS, namely: 1) setting and facilitating engagement by education and confidence-building; 2) increasing the importance placed on QoL and patient concerns through patient-reported outcomes (PROs); 3) providing credible sources of accurate information; 4) encouraging treatment adherence through engagement; and 5) empowering through a sense of responsibility. Group members independently researched and contributed examples of patient engagement strategies from several countries and examined interventions that have worked well in areas of patient engagement in MS, and other chronic illnesses. The group presents their perspective on these programs, discusses the barriers to achieving patient engagement, and suggests practical strategies for overcoming these barriers. With an understanding of the issues that influence patient engagement in MS, we can start to investigate ways to enhance engagement and subsequent health outcomes. Engaging patients involves a broad, multidisciplinary approach.