Assessing treatment outcomes in multiple sclerosis trials and in the clinical setting

Increasing numbers of drugs are being developed for the treatment of multiple sclerosis (MS). Measurement of relevant outcomes is key for assessing the efficacy of new drugs in clinical trials and for monitoring responses to disease-modifying drugs in individual patients. Most outcomes used in trial and clinical settings reflect either clinical or neuroimaging aspects of MS (such as relapse and accrual of disability or the presence of visible inflammation and brain tissue loss, respectively). However, most measures employed in clinical trials to assess treatment effects are not used in routine practice. In clinical trials, the appropriate choice of outcome measures is crucial because the results determine whether a drug is considered effective and therefore worthy of further development; in the clinic, outcome measures can guide treatment decisions, such as choosing a first-line disease-modifying drug or escalating to second-line treatment. This Review discusses clinical, neuroimaging and composite outcome measures for MS, including patient-reported outcome measures, used in both trials and the clinical setting. Its aim is to help clinicians and researchers navigate through the multiple options encountered when choosing an outcome measure. Barriers and limitations that need to be overcome to translate trial outcome measures into the clinical setting are also discussed

Defining and scoring response to IFN-β in multiple sclerosis

The advent of a large number of new therapies for multiple sclerosis (MS) warrants the development of tools that enable selection of the best treatment option for each new patient with MS. Evidence from clinical trials clearly supports the efficacy of IFN-β for the treatment of MS, but few factors that predict a response to this drug in individual patients have emerged. This deficit might be due, at least in part, to the lack of a standardized definition of the clinical outcomes that signify improvement or worsening of the disease. MRI markers and clinical relapses have been the most widely studied short-term factors to predict long-term response to IFN-β, although the results are conflicting. Recently, integrated strategies combining MRI and clinical markers in scoring systems have provided a potentially useful approach for the management of patients with MS. In this Review, we focus on the many definitions of clinical response to IFN-β and explore the markers that can be used to predict this response. We also highlight advantages and limitations of the existing scoring systems in light of future expansion of these models to biological markers and to other classes of emerging therapies for MS