Clinical Trial Design to Facilitate Biomarker Discovery

The role of clinical trials to generate knowledge about IBD treatments is very important. Endpoints have shifted from mainly subjective measures such as clinical disease activity, to patient reported outcomes in combination with objective measurements such as mucosal improvement. Even though therapeutic options for treatment of IBD are increasing, with many new therapeutic antibodies and small molecules under active investigation, treatment failure and withdrawal rates in clinical trials vary from 30–50%. Aside from side effects, primary non-response and secondary loss of response are possible reasons for discontinuation. More individualized treatment based on optimized dosing strategies and phenotypic and/or biological markers will probably lead to higher success rates. To move towards more individualized treatment in IBD, identification of biomarkers that can predict response to treatment would be valuable. Data and materials collected in clinical trials are a valuable source for identification of predictive biomarkers or development of pharmacokinetic models. By using a dashboard system with an incorporated pharmacokinetic model, the exact medication dose a patient should receive and the exact date it should be given to optimize drug exposure can be calculated. Pharmacokinetic analyses are increasingly used both in daily clinical practice as well as in clinical trials to individualize and thereby optimize medical treatment.