Application of Comparative Effectiveness Research in Evaluation of Treatments for Type 2 Diabetes Mellitus

Abstract Comparative effectiveness research (CER) is a relatively new strategy in drug development and healthcare designed to aid consumers, clinicians, purchasers, and policy makers in determining the best treatment options for individual patients. While defined in scope of clinical research, CER has the potential to be applied to other outcomes including economic evaluation of treatments and their potential impact on overall healthcare utilization by using all available data from clinical trials, systematic reviews, meta-analyses, and observational studies. Whereas clinical trials have mainly compared new agents with placebo (either alone or added to background therapy), CER compares new treatments head-to-head with active comparators to determine a patient's best available options. Because of the emphasis on the patient, and its potential to identify the best treatment options and produce substantial cost savings, CER is likely to be an integral part of American healthcare reform. The Affordable Care Act's emphasis on CER should benefit patients with type 2 diabetes (T2DM), a major chronic disease that affects 26 million Americans. The most recent innovations in marketed T2DM therapies include 2 classes of drugs focused on the incretin system: glucagon-like peptide-1 (GLP-1) receptor agonists and dipeptidyl peptidase-4 (DPP-4) inhibitors. This article reviews available CER among the incretin-based agents and for older T2DM therapies. CER is expected to open new avenues for research to clarify best practices in the treatment of T2DM, as well as possibly reduce treatment costs and improve the overall quality of public health.