Rieckmann et al. Achieving patient engagement in multiple sclerosis: A perspective from the multiple sclerosis in the 21st Century Steering Group. Mult Scler Relat Disord. 2015; 4:202-218.
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.
So is the Blog just the ticket as a Blockbuster Drug?
The “MS in the 21st Century” initiative was established with the purpose of (1) defining how multiple sclerosis (MS) treatment and standards of care should look in the 21st century; (2) developing a minimum standard of care across the world; and (3) motivating the broad MS community to align standards of care and challenge the current treatment paradigm.
The aim was to develop a consensus statement to reach and influence the broader MS community. An expert steering group from Europe and Canada—consisting of neurologists, patient advocates, a pharmacoepidemiologist/pharmacoeconomist, and representatives from national MS centers—participated in a series of workshop-driven meetings between February 2011 and 2012. After three phases of discussions, the steering group identified that the overall vision for future care of MS should be full access to personalized treatment, with reimbursement, to achieve freedom from disease. They constructed seven overall principles that support this vision: personalized care, patient engagement, commitment to research, regulatory body education and reimbursement issues, new endpoints in clinical trials, more therapy options, and MS centres of excellence. This consensus statement outlines the key aspects of the seven principles that need to be addressed. The “MS in the 21st Century Steering Group” hopes that this consensus statement acts as a call to action for healthcare providers and decision-makers to address simultaneously the overarching principles that will guide patient management in order to improve outcomes for people with MS.
To realize our vision of full access to personalized treatment, with reimbursement, to achieve freedom from disease, we call upon healthcare providers and decision-makers to address simultaneously the following overarching principles that will guide patient management, in order to improve outcomes for people with MS.
We need personalized treatments for all types of MS . This means developing new approaches that incorporate a wide range of pharmacological and non-pharmacological strategies that focus on the needs of people living with the disease as individuals, and aim to reduce disease activity, slow disability progression and improve management of symptoms MS care requires diverse therapies and strategies to address the complexity of the disease’s considerable symptoms and challenges . Governments, healthcare decision-makers, and healthcare systems must be prepared to see MS patients as requiring interventions beyond greater access to disease-modifying agents alone, especially for those with progressive forms of MS.The best treatment regimen is often specific to the patient and best achieved through continuity of care with a team of physicians and allied professionals. Healthcare decision-makers must realize that this outcome is often compromised in modern hospitals, where, in the name of efficiency, patients will generally see a sequence of different doctors and may receive increasingly fragmented care. To overcome this barrier, we call on healthcare policy-makers to prioritize the development of centers of excellence for improved access to personalized treatment and to empower MS nurses as central coordinators for patient care.Patients also have the right to be informed about, and involved in, all decisions regarding their treatment, including early therapy options. All available therapies and their respective risks and benefits should be communicated to patients by their physicians, and patients should also have access to other reliable, independent sources to ensure that they are empowered to make informed decisions. In addition, affording patients psychological and social support as part of their treatment package is likely to ensure the greatest possible mitigation of the potential financial, social, and psychosocial burdens associated with MS.
Patients should be engaged as advocates. They should be empowered to drive negotiations with regulators and payers In addition, patient organizations should work more closely with clinicians, industry, and regulatory bodies to petition for and secure research funds.
Commitment to research We need to embed a culture of research in all aspects of MS care. The continued progress of basic research is currently under threat due to reduced charity income and governmental funding. However, research across all domains is key for a better understanding of disease mechanisms, which ultimately leads to more effective new treatment options (pharmacological and non-pharmacological), including therapies for progressive forms of MS. We recommend the development of an international network of MS centers of excellence (see below the MS centers of excellence) as a platform for establishing research networks and leveraging funding. In addition, without compromising patient safety, we call upon regulators to re-evaluate the existing European Commission (EC) requirement for investigator-led hypothesis-generating studies to meet the same standards as industry-funded clinical trials, as this criterion has severely limited the scope of such investigator-led studies.
Regulatory body education and reimbursement issues
The authors believe that patient groups, clinicians, and industry need to have greater access to regulatory bodies at a national and local level in order to: Improve the regulator’s understanding of the complexities associated with the treatment and care of MS patients Align perceptions of the risks of the disease and its treatment. The introduction of a conditional license, allowing several thousand patients to be treated for a number of years, would allow safety data to be formally gathered from people with MS who are willing to take greater risks and be exposed to greater uncertainty. Regulatory bodies need to consider how escalating regulatory requirements, including increasing demands for long-term safety data, could be major drivers for spiraling development costs. This leads to increasing drug prices, limited innovation, and delays in getting promising drugs to patients. Reimbursement agencies should consider the substantial indirect costs associated with MS and the related burden to society in their calculation of cost:benefit ratios.
New endpoints in clinical trials
Clinical trials in MS are still largely based on single-parameter measures of disease relapses or disability progression . Other parameters, such as subclinical inflammatory activity, neuroprotection, lesion burden detected by MRI, health economics, cognitive impairment, and fatigue are regarded only as secondary endpoints or are rarely measured at all. Employing the use of single-parameter measures in clinical trials can fail to measure the effect of treatment on the actual disease process, and these strategies only partly reflect real-world treatment scenarios. Developing better patient- and physician-reported tools are needed for the assessment of these parameters, and using those within composite endpoints may, therefore, improve measures of treatment efficacy. In fact, a number of groups have recently used composite endpoints in phase 3 clinical trials with success. New clinical trial designs and endpoints that enable effective treatment development and evaluation to achieve relevant benefits are needed. Such endpoints should include patient-reported measures, clinical and functional assessments, and biomarkers. We encourage the development of initiatives involving regulatory authorities, expert groups, and industry, which will amalgamate these disparate elements into new composite endpoints/scales.
More therapy options
Although there are already multiple pharmacological treatment options available for MS, the high costs of these treatments may limit patient access. Governments, regulators, funding agencies, clinicians, the pharmaceutical industry, and patient groups need to work together to develop strategies to deliver more cost-effective medicines. They also need to widen the focus of research to ensure the continuous development of better therapy options with improved efficacy, safety, and tolerability profiles. Patients should have greater access to comprehensive care regimens that include symptomatic care, rehabilitation, and psychological support alongside pharmacological solutions. Non-responding patients and those with aggressive disease should be allowed access to experimental treatment options in well-controlled programs. We petition regulatory bodies and funders to provide this access. In addition, we ask regulators and funders to consider the relevance of the placebo arm and the assimilation of more long-term data on the therapy options available. In this regard, we support the European Commission’s consideration for a patient registry.
MS centres of excellence
We need a network of dedicated MS centres of excellence that meet a set of predetermined minimum standards and demonstrate a will to contribute to research and to share resources. This is to improve diagnosis and treatment, as well as to provide optimum patient support.
CoI: Prof G is a coauthor on this paper