Neuroepidemiology. Part II: Assessment of therapeutic trials.
The gold standard for therapeutic trials is the randomized, double-blind, placebo-controlled study design. Lack of blindness and placebo makes tenuous the attribution of results to the specific agent; lack of randomization between concurrent and comparable groups makes it impossible. The chi-square test of the null hypothesis of no difference between treatment groups is the best method of assessment, as no overall rating system used in neurology is a true numerical scale but at best a rank-order scale. If quantitation of results is desired, a nonparametric rank-order test is necessary. Results over time can be assessed with a logrank (life-table) test for single events as end point, and by chi-square or rank-order tests for multiple events among individual patients.
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D002986:Clinical Trials as Topic / Q000379:methods / Q000592:standards*; D004185:Disability Evaluation; D006801:Humans; D009103:Multiple Sclerosis / Q000503:physiopathology; D009422:Nervous System Diseases / Q000453:epidemiology / Q000503:physiopathology*; D009460:Neurologic Examination; D012107:Research Design; D013997:Time Factors; D014481:United States