In a special feature published in the journal American Health & Drug Benefits, authors Stanton R. Mehr, President of SM Health Communications, and Marj P. Zimmerman, President of RxDirections, discuss the many unmet medical needs multiple sclerosis (MS) patients still face in dealing with the disease.
Over the past few decades, research focusing mainly on the prevention and reduction of relapses in the relapsing-remitting form of MS (RRMS) have led to some therapeutic advances. But while preventing disease relapse is indeed an important treatment goal, high levels of disability is still a major obstacle in the daily lives of MS patients.
The feature, entitled “Reviewing the Unmet Needs of Patients with Multiple Sclerosis,“ plunges head-first into the rather extensive body of research investigating what MS patients still lack in terms of treatments that can make a different in their quality of life.
Delaying progression and developing better treatments for progressive MS
Progressive MS treatment and delay of disease progression have not received the same research focus as RRMS. Further delaying progression and developing better treatments for progressive MS is, therefore, one of the main points listed in this overview. A new agent, ocrelizumab — which is due to be reviewed by the FDA in 2016 — was found to decrease disability in progressive MS patients in a large clinical trial. Patients and clinicians alike hope that this drug might prove to be an advance in the treatment of progressive MS.
Researchers argue that if it were possible to protect neurons from the demyelination occurring in MS, it would translate into severely slowed disease progression and reduced disability. Research to this point has focused predominantly on potential mechanisms of neuroprotection. While current immunomodulating drugs might provide a certain level of protection against demyelination, the authors concluded that the link between these drugs and potential neuroprotection has not been sufficiently studied.
Delaying or avoiding disability
Studies into neuroprotection would also offer insights into how to delay or avoid disability — a major concern for all MS patients. Patients typically receive an MS diagnosis during the most productive years of their life and live with the disease for another 30-40 years.
Several studies show that psychological disability is not nearly addressed by clinicians as often as the patients would wish, and a large proportion of patients cite psychological factors as their greatest unmet need. Such factors undoubtedly also affect caregivers of MS patients, who are likely to receive equally little psychological support.
Physical disability has been shown to reduce employment by up to 80 percent as early as five years after diagnosis, and current medications contribute little in terms of improving physical disability. In addition to reduced mobility, many patients suffer from dysphagia (difficulty in swallowing), speech problems, and decreased vision.
Obtaining better measures of functional outcome
While MS clinicians are aware of the limitations of the Expanded Disability Status Scale (EDSS) — based on neurological evaluations — it is still widely used to assess disease progression. Although other outcome measures have been recommended, the International Advisory Committee on Clinical Trials in Multiple Sclerosis recommends that the EDSS continue to be used because it is widely accepted by regulators. However, they also encourage physicians to complement clinical data with other measurements, particularly focusing on patient-reported outcomes.
Reducing active symptoms more effectively
Current symptomatic treatment during acute disease exacerbation is not perceived as adequate by MS patients. The authors believe that more can be done to research and develop new therapeutic options for managing MS symptoms in RRMS.
Preventing side effects and improving adherence to medications
MS medications are associated with a host of adverse effects, ranging from nausea to serious complications such as progressive multifocal leukoencephalopathy (PML). Such side effects are severely affecting the lives of MS patients and contributing to disability. Because of the frequent occurrence of adverse effects, adherence to medications is often low, specifically in patients with secondary progressive MS. Improving adherence is, therefore, included in the overview of MS patients’ needs.
Identifying biomarkers and predictive tools
While magnetic resonance imaging (MRI) can be used to predict disease progression and future disability, the use of other biomarkers for disease progression and treatment response are not well-developed. Neither are diagnostic biomarkers indicating MS risk. Some progress has been made with markers improving the safety of drug therapy, reducing the risk of PML.
There is no “one drug fits all” in MS. Some specialty drugs, often expensive commodities, fail to produce a response in certain patients. Predictive tests of treatment response, based on the patients’ clinical presentation, are therefore on the wish-list of both patients and clinicians.
The authors concluded that despite the attention given to MS in previous years, many unmet needs still remain, especially regarding effective therapeutics, disability prevention, and proper outcome measures.