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

摘要: Poorly designed trials of therapy for multiple sclerosis (MS) can waste time and money, may lead either to false hopes or the overlooking a potentially effective treatment. A well-designed trial well develop useful scientific information even if putative fails show any therapeutic effect. The diagnosis, clinical course, definitions stages MS are discussed as they relate therapy. goals such include favorable modification an exacerbation, prevention future exacerbations, treatment progressive stage, improvement function in stable-deficit stage. There should be orderly progression from small preliminary modest pilot and, when indicated, full trial. All types require careful organization management, appropriate selection patients, properly planned recorded observations. contrast-how new will evaluated-provides essential structure hypothesis being examined, contrast, observations made govern form analysis nature interpretations. Each goal requires that specific strategies design considerations applied preliminary, pilot, trials.