title: Design issues and outcomes in IBD clinical trials.
abstract: Successful clinical trials in inflammatory bowel disease are based on precise definitions of study populations, standardized and well-defined interventions, reliable indices of disease activity, and clearly stipulated outcome measures. Interpretation of research results is often complicated by the differentiation of goals of therapy (remission induction and maintenance, quality of life) and the multitude of patient subsets. Choosing the correct patient subtype to enroll in a clinical trial is critical to the results of a study, its conclusions, and its applicability to the clinical setting. Validated, easy-to-use disease activity indices allow interpretation of results across trials. The use of biomarkers as surrogate clinical endpoints provides reproducibility, ease of statistical handling as a continuous variable, and consistent measurement of response to treatment. Despite these potential advantages, biomarkers of disease activity lack specificity and will need to be validated against clinically meaningful outcomes. Careful subject selection, standardization of disease activity indices, and precise outcome measurement are the keys to continued improvement of the inflammatory bowel disease research process.
authors.affiliation: Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
authors.forename: Bruce E
authors.initials: BE
authors.lastname: Sands
authors.forename: Maria T
authors.initials: MT
authors.lastname: Abreu
authors.forename: George D
authors.initials: GD
authors.lastname: Ferry
authors.forename: Anne M
authors.initials: AM
authors.lastname: Griffiths
authors.forename: Stephen B
authors.initials: SB
authors.lastname: Hanauer
authors.forename: Kim L
authors.initials: KL
authors.lastname: Isaacs
authors.forename: James D
authors.initials: JD
authors.lastname: Lewis
authors.forename: William J
authors.initials: WJ
authors.lastname: Sandborn
authors.forename: A Hillary
authors.initials: AH
authors.lastname: Steinhart
mesh_terms: D015415:Biomarkers / Q000032:analysis; D002986:Clinical Trials as Topic; D023381:Endpoint Determination; D006801:Humans; D015212:Inflammatory Bowel Diseases / Q000188:drug therapy*; D010641:Phenotype; D011788:Quality of Life; D012107:Research Design; D016896:Treatment Outcome
chemical_list: D015415:Biomarkers