ASSESSING OUTCOME IN RANDOMIZED CLINICAL-TRIALS - INFLAMMATORY BOWEL-DISEASE

Methodological principles or standards to assess the quality of a clin ical trial must be simple and user-friendly if we expect busy clinicia ns to adopt them. When assessing the success or failure of a new treat ment in inflammatory bowel disease, the patient characteristics, thera peutic strategy, outcome assessment and interpretation of results are unique to the disease and the study. Because inflammatory bowel diseas e rarely is fatal, we use surrogate markers, such as disease activity index, endoscopic appearance, histology, blood tests, tissue markers, need for other medications and quality of life scores, to assess treat ment efficacy. Primary and secondary outcomes must be identified and u niformly evaluated to ensure unbiased objective assessment. Careful a priori definition of outcome events is essential. The statistical anal ysis depends on the data type which comprise the outcome events and th e study design. Both intention to treat as well as efficacy analyses s hould be performed. Interpretation of results should address both clin ical and statistical importance.