Outcome measures in multiple sclerosis clinical trials.
Multiple sclerosis remains incurable. The urgency of this problem, together with the need to test an ever increasing number of promising therapeutic agents emerging from animal studies, has renewed interest in both the application and the development of efficient outcome measures for treatment trials. The selection and usage of instruments used to detect outcomes is probably the most important issue in treatment trial design. Thus, familiarity with them is essential not only for researchers, but also for clinicians who wish to interpret trial results critically. This chapter begins with a discussion of the properties of an ideal outcome instrument, then reviews the strengths and limitations of existing clinical, radiological and laboratory outcome measures. With this knowledge, the current consensus regarding appropriate outcome measure selection, as well as recommendations and future perspectives in trial design, are discussed.
Andersson
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PB
Department of Neurology, Stanford University Medical Center, CA 94305-5235, USA.
Goodkin
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DE
D015415:Biomarkers; D002986:Clinical Trials as Topic / Q000379:methods* / Q000592:standards; D004185:Disability Evaluation; D018450:Disease Progression; D006805:Human Experimentation; D006801:Humans; D008279:Magnetic Resonance Imaging; D009103:Multiple Sclerosis / Q000175:diagnosis / Q000503:physiopathology / Q000628:therapy*; D009483:Neuropsychological Tests; D017063:Outcome Assessment, Health Care / Q000379:methods / Q000592:standards*; D012107:Research Design / Q000592:standards*; D012720:Severity of Illness Index
D015415:Biomarkers