Comparative Effectiveness Research: A Pharmaceutical Industry Perspective on Outlook, Dilemmas, and Controversies

Abstract

Comparative effectiveness research (CER) is the process of examining the performance of various medical treatments against each other. Many factors can affect how CER is performed and what conclusions are derived from any given study. Though the gold standard in health-care research remains the randomized controlled trial, CER studies that utilize real-world evidence can be valuable, as long as such studies are fit for purpose, have a rigorous methodology, and are easily interpretable and sources of bias have been minimized and disclosed. Though technological advances make it increasingly easier to perform sophisticated comparative analyses using ever-larger and increasingly detailed datasets, adoption and dissemination of CER by the US pharmaceutical industry has been surprisingly slow. This may be due to myriad of factors including regulatory and legal pitfalls, economic incentives, cultural influences, and public perceptions. Nevertheless, in an environment where both technological and economic pressures require smarter, less-resource intense ways of understanding the value and benefit of all existing treatments, CER has tremendous potential to improve decision making through its evidence-based approach to treatment choices. Alternative ways of conducting, interpreting, and disseminating CER should be a priority for the industry.