Recommendations for the Definition of Clinical Responder in Insulin Preservation Studies
Clinical Trial News Monday, May 5th, 2014Diabetes: April 10, 2014
Clinical responder studies should contribute to the translation of effective treatments and interventions to the clinic. Since ultimately this translation will involve regulatory approval, we recommend that clinical trials pre-specify a responder definition that can be assessed against the requirements and suggestions of regulatory agencies. In this paper we propose a clinical responder definition to specifically assist researchers and regulatory agencies interpret the clinical importance of statistically significant findings for studies of interventions intended to preserve β-cell function in newly diagnosed Type 1 diabetes. We focus on studies of six-month β-cell preservation in type I diabetes as measured by 2-hour stimulated C-peptide. We introduce criteria (bias, reliability and external validity) for the assessment of responder definitions to ensure they meet FDA and European Medicines Agency guidelines. Read More
