Regulatory Benefit-Risk Assessment and Comparative Effectiveness Research: Strangers, Bedfellows or Strange Bedfellows?

Over the past 5 years, we have witnessed growing interest in both comparative effectiveness research (CER) and regulatory benefit-risk assessment (BRA). Both deal with benefits and harms, although at different stages of the product lifecycle. There are growing pressures for a more systematic and quantitative approach to regulatory BRA. However, there is also a need for CER - beyond the evidence that can reasonably be generated during pre-launch product development. Important regulatory and policy questions include the following: What would be a level playing field across disease areas and companies? Who should bear the costs of these studies? What role can benefit-risk modelling play? What is the value of research and how is it related to the prevalence of disease? What is the relationship between uncertainty and the value of evidence? We need to recognize the lifecycle nature of evidence generation, moving from the regulatory setting to the real world and affecting potentially hundreds of thousands, or even millions, of patients worldwide. We need to emphasize not only the public goods nature of information embedded in innovations, but also that it is global. Finally, we need to more systematically explore the benefits and costs of gathering further information - the value of research - recognizing that doing this requires a model or methodology, which we have, for systematically appraising our current state of knowledge and what could be gained from further research. All said, it would seem that BRA and CER should be neither strangers nor strange bedfellows, but may need to be coaxed into being bedfellows.