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The design of clinical studies to assess therapeutic efficacy in multiple sclerosis

Department of Neurology (Emeritus), Mayo Clinic, Rochester, Minnesota, and the Department of Neurosciences, University of California San Diego, San Diego, California (Dr. Brown); Clinical Epidemiology Branch, National Cancer Institute, Bethesda, Maryland (Dr. Beebe); Departments of Neurology and Community Medicine, Georgetown University and Neurology Service, Veterans Administration Hospital, Washington, D.C. (Dr. Kurtzke); Department of Neurology, Medical School and Division of Biometry, School of Public Health, University of Minnesota, Minneapolis, Minnesota (Dr. Loewenson); Department of Neurology, University of Pennsylvania, Philadelphia, Pennsylvania (Dr. Silberberg); Department of Neurology, University of California Los Angeles and Department of Neurology, Wadsworth Veterans Administration Hospital, Los Angeles, California (Dr. Tourtellotte).

Poorly designed trials of therapy for multiple sclerosis (MS) can waste time and money, and may lead either to false hopes or to the overlooking of a potentially effective treatment. A well-designed trial may well develop useful scientific information even if the putative therapy fails to show any therapeutic effect.

The diagnosis, clinical course, and definitions of the stages of MS are discussed as they relate to trials of therapy. The goals of such trials include favorable modification of an exacerbation, favorable modification or prevention of future exacerbations, effective treatment of the progressive stage, and improvement of function in the stable-deficit stage. There should be an orderly progression from a small preliminary trial to a modest pilot trial and, when indicated, a full trial. All types of trials require careful organization and management, appropriate selection of patients, and properly planned and recorded observations.

The treatment contrast-how the new treatment will be evaluated-provides the essential structure of the trial.

The hypothesis being examined, the treatment contrast, and the observations being made in the designed clinical trial will govern the form of the analysis and the nature of the interpretations.

Each goal requires that specific strategies and design considerations be applied to preliminary, pilot, and full trials.

Requests for reprints should be addressed to Research Programs Department, NMSS, 205 East 42nd Street, New York, NY 10017.

This study is supported by an appropriation from the National Multiple Sclerosis Society (NMSS)

The paper reflects the efforts of an Ad Hoc Working Group on the Design of Clinical Studies to Assess Therapeutic Eficacy in Multiple Sclerosis appointed by Research Programs Committee and Research Staff of NMSS.

Accepted for publication February 21, 1979.

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