Design of Treatment Trials for Disorders of Gut-Brain Interaction.

Abstract

Rigorous treatment trials have resulted in substantial therapeutic advances in disorders of gut-brain interaction (DBGI) over the past several decades. However, much of this work has been conducted in adults with irritable bowel syndrome, and some of the challenges of DGBI trials (eg, symptom-based diagnoses and outcomes) have experienced more limited progress, especially for less common DGBI. Randomized, placebo-controlled trials remain the time-tested gold standard for DGBI, although more recent pragmatic trials have demonstrated real-world efficacy. Moreover, treatment trials have expanded into dietary interventions and brain-gut behavioral therapies, now with greater guidance and recognition of the nuances of these nondrug trials. Development of validated patient-reported outcome measures and adherence to Good Clinical Practice standards will yield novel DGBI therapies and more sophisticated treatment strategies for the management of these complex disorders. This article summarizes best practice, methodology, conduct, and reporting of treatment trials in DGBI as well as regulatory guidance and recent advances in trial design.