Current issues in the design of clinical trials in IBD.

Abstract

Although the randomized controlled trial has become the standard for regulatory approval of new drugs and devices in inflammatory bowel disease (IBD), the components of effective trial design and implementation are still evolving. While induction and maintenance of remission are the ultimate goals in the treatment of IBD, the conduct of trials intended to measure these outcomes has varied substantially over time, as have the definitions of disease remission. Significant progress has been made in recent years towards understanding patient- and disease-related factors that are essential considerations in clinical trial design. However, questions remain regarding the best methods for assessing disease activity, and importantly for establishing trial end points that are clinically relevant and likely to affect meaningful long-term improvements in disease outcomes. This chapter will discuss the current 'best methods' in IBD trial design and introduce potential future concepts for improving the efficiency of clinical trials as well as their utility to inform clinical practice management.